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Tytuł :
Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial.
Autorzy :
Boorjian SA; Department of Urology, Mayo Clinic, Rochester, MN, USA.
Alemozaffar M; Department of Urology, Kaiser Permanente Los Angeles, Los Angeles, CA, USA.
Konety BR; Department of Urology, University of Minnesota, Minneapolis, MN, USA.
Shore ND; Carolina Urologic Research Center, Myrtle Beach, SC, USA.
Gomella LG; Department of Urology, Sidney Kimmel Cancer Center, Thomas Jefferson University, Philadelphia, PA, USA.
Kamat AM; Department of Urology, University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Bivalacqua TJ; Department of Urology, The James Buchanan Brady Urological Institute, Johns Hopkins School of Medicine, Baltimore, MD, USA.
Montgomery JS; Department of Urology, University of Michigan, Ann Arbor, MI, USA.
Lerner SP; Scott Department of Urology, Dan L Duncan Cancer Center, Baylor College of Medicine, Houston, TX, USA.
Busby JE; Cancer Centers of the Carolinas, Greenville Hospital System, Greenville, SC, USA.
Poch M; Department of GU Oncology, H Lee Moffitt Cancer Center & Research Institute, Tampa, FL, USA.
Crispen PL; Department of Urology, University of Florida, Gainesville, FL, USA.
Steinberg GD; Department of Urology, New York University Langone Health, New York, NY, USA.
Schuckman AK; USC Institute of Urology, Norris Comprehensive Cancer Center, University of Southern California, Los Angeles, CA, USA.
Downs TM; Department of Urology, University of Wisconsin, Madison, WI, USA.
Svatek RS; Department of Urology, University of Texas Health Science Center at San Antonio, San Antonio, TX, USA.
Mashni J Jr; Banner MD Anderson Cancer Center, Gilbert, AZ, USA.
Lane BR; Division of Urology, Spectrum Health, Michigan State University College of Human Medicine, Grand Rapids, MI, USA.
Guzzo TJ; Division of Urology, University of Pennsylvania, Philadelphia, PA, USA.
Bratslavsky G; Department of Urology, SUNY Upstate Medical University, Syracuse, NY, USA.
Karsh LI; The Urology Center of Colorado, Denver, CO, USA.
Woods ME; Department of Urology, University of North Carolina, Chapel Hill, NC, USA.
Brown G; New Jersey Urology, Bloomfield, NJ, USA.
Canter D; Ochsner Health System, Jefferson, LA, USA.
Luchey A; West Virginia University Cancer Institute, Morgantown, WV, USA.
Lotan Y; Department of Urology, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Krupski T; Department of Urology, University of Virginia Cancer Center, Charlottesville, VA, USA.
Inman BA; Division of Urology, Department of Surgery, Duke University School of Medicine, Durham, NC, USA.
Williams MB; Urology of Virginia, Virginia Beach, VA, USA.
Cookson MS; Department of Urology, University of Oklahoma Health Sciences Center, Oklahoma City, OK, USA.
Keegan KA; Department of Urology, Vanderbilt University Medical Center, Nashville, TN, USA.
Andriole GL Jr; Washington University School of Medicine in St Louis, St Louis, MO, USA.
Sankin AI; Department of Urology, Montefiore Medical Center, Bronx, NY, USA.
Boyd A; Alan Boyd Consultants, Crewe, UK.
O'Donnell MA; Department of Urology, University of Iowa, Iowa City, IA, USA.
Sawutz D; FKD Therapies Oy, Kuopio, Finland.
Philipson R; Trizell, Chinnor, Oxon, UK.
Coll R; Trizell, Chinnor, Oxon, UK.
Narayan VM; Department of Urology, University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Treasure FP; Peter Treasure Statistical Services, King's Lynn, UK.
Yla-Herttuala S; AI Virtanen Institute University of Eastern Finland and Science Service Center and Gene Therapy Unit, Kuopio, Finland.
Parker NR; AI Virtanen Institute University of Eastern Finland and Science Service Center and Gene Therapy Unit, Kuopio, Finland.
Dinney CPN; Department of Urology, University of Texas MD Anderson Cancer Center, Houston, TX, USA. Electronic address: .
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Źródło :
The Lancet. Oncology [Lancet Oncol] 2021 Jan; Vol. 22 (1), pp. 107-117. Date of Electronic Publication: 2020 Nov 27.
Typ publikacji :
Clinical Trial, Phase III; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't
MeSH Terms :
Drug Resistance, Neoplasm*
Genetic Therapy*/adverse effects
Genetic Therapy*/mortality
Genetic Vectors*
Adenoviridae/*genetics
BCG Vaccine/*administration & dosage
Carcinoma in Situ/*therapy
Interferon alpha-2/*genetics
Urinary Bladder Neoplasms/*therapy
Administration, Intravesical ; Aged ; BCG Vaccine/adverse effects ; Carcinoma in Situ/genetics ; Carcinoma in Situ/mortality ; Carcinoma in Situ/pathology ; Disease Progression ; Female ; Humans ; Male ; Middle Aged ; Neoplasm Grading ; Neoplasm Invasiveness ; Neoplasm Recurrence, Local ; Time Factors ; Treatment Outcome ; United States ; Urinary Bladder Neoplasms/genetics ; Urinary Bladder Neoplasms/mortality ; Urinary Bladder Neoplasms/pathology
Czasopismo naukowe
Tytuł :
Recent trends in cancer therapy: A review on the current state of gene delivery.
Autorzy :
Yahya EB; School of Industrial Technology, Universiti Sains Malaysia, Penang 11800, Malaysia. Electronic address: .
Alqadhi AM; Faculty of Medicine, Al Mergib University, Al Khums, Libya.
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Źródło :
Life sciences [Life Sci] 2021 Mar 15; Vol. 269, pp. 119087. Date of Electronic Publication: 2021 Jan 19.
Typ publikacji :
Journal Article; Review
MeSH Terms :
CRISPR-Cas Systems*
Gene Editing*
Gene Transfer Techniques*
Genetic Therapy*
Neoplasms/*therapy
Animals ; Humans ; Neoplasms/genetics
Czasopismo naukowe
Tytuł :
CRISPR-cas9 genome editing delivery systems for targeted cancer therapy.
Autorzy :
Ghaemi A; Pharmaceutical Research Center, Pharmaceutical Technology Institute, Mashhad University of Medical Sciences, Mashhad, Iran; Department of Pharmaceutical Biotechnology, School of Pharmacy, Mashhad University of Medical Sciences, Mashhad, Iran.
Bagheri E; Pharmaceutical Research Center, Pharmaceutical Technology Institute, Mashhad University of Medical Sciences, Mashhad, Iran; Department of Pharmaceutical Biotechnology, School of Pharmacy, Mashhad University of Medical Sciences, Mashhad, Iran.
Abnous K; Pharmaceutical Research Center, Pharmaceutical Technology Institute, Mashhad University of Medical Sciences, Mashhad, Iran; Department of Medicinal Chemistry, School of Pharmacy, Mashhad University of Medical Sciences, Mashhad, Iran.
Taghdisi SM; Department of Pharmaceutical Biotechnology, School of Pharmacy, Mashhad University of Medical Sciences, Mashhad, Iran; Targeted Drug Delivery Research Center, Pharmaceutical Technology Institute, Mashhad University of Medical Sciences, Mashhad, Iran.
Ramezani M; Pharmaceutical Research Center, Pharmaceutical Technology Institute, Mashhad University of Medical Sciences, Mashhad, Iran; Department of Pharmaceutical Biotechnology, School of Pharmacy, Mashhad University of Medical Sciences, Mashhad, Iran. Electronic address: .
Alibolandi M; Pharmaceutical Research Center, Pharmaceutical Technology Institute, Mashhad University of Medical Sciences, Mashhad, Iran; Department of Pharmaceutical Biotechnology, School of Pharmacy, Mashhad University of Medical Sciences, Mashhad, Iran. Electronic address: .
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Źródło :
Life sciences [Life Sci] 2021 Feb 15; Vol. 267, pp. 118969. Date of Electronic Publication: 2020 Dec 29.
Typ publikacji :
Journal Article; Review
MeSH Terms :
CRISPR-Cas Systems/*physiology
Genetic Therapy/*trends
Neoplasms/*therapy
CRISPR-Cas Systems/genetics ; Clustered Regularly Interspaced Short Palindromic Repeats ; DNA/genetics ; Gene Editing/methods ; Genetic Therapy/methods ; Genome ; Humans ; Neoplasms/genetics
Czasopismo naukowe
Tytuł :
Review on mechanistic strategy of gene therapy in the treatment of disease.
Autorzy :
Alnasser SM; Department of Pharmacology and Toxicology, College of Pharmacy, Qassim University, Unaizah, Saudi Arabia.
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Źródło :
Gene [Gene] 2021 Feb 15; Vol. 769, pp. 145246. Date of Electronic Publication: 2020 Oct 22.
Typ publikacji :
Journal Article; Review
MeSH Terms :
Genetic Therapy*
Gene Transfer Techniques ; Genetic Vectors ; Humans
Czasopismo naukowe
Tytuł :
Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies.
Autorzy :
Sheikh O; Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta , Edmonton, Canada.
Yokota T; Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta , Edmonton, Canada.
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Źródło :
Expert opinion on investigational drugs [Expert Opin Investig Drugs] 2021 Feb; Vol. 30 (2), pp. 167-176. Date of Electronic Publication: 2021 Jan 06.
Typ publikacji :
Journal Article; Review
MeSH Terms :
Genetic Therapy*/adverse effects
Cell-Penetrating Peptides/*therapeutic use
Dystrophin/*genetics
Muscular Dystrophy, Duchenne/*therapy
Oligonucleotides/*therapeutic use
Animals ; Cell-Penetrating Peptides/adverse effects ; Codon, Terminator ; Drug Development ; Drugs, Investigational/adverse effects ; Drugs, Investigational/therapeutic use ; Exons ; Gene Expression Regulation ; Genetic Predisposition to Disease ; Humans ; Muscular Dystrophy, Duchenne/diagnosis ; Muscular Dystrophy, Duchenne/genetics ; Mutation ; Oligonucleotides/adverse effects ; Steroids/adverse effects ; Steroids/therapeutic use ; Treatment Outcome
Czasopismo naukowe
Tytuł :
The functional characteristics of optogenetic gene therapy for vision restoration.
Autorzy :
Lindner M; The Nuffield Laboratory of Ophthalmology, Sleep and Circadian Neuroscience Institute, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK. .; Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK. .; Institute of Physiology and Pathophysiology, Department of Neurophysiology, Philipps University, Marburg, Germany. .
Gilhooley MJ; The Nuffield Laboratory of Ophthalmology, Sleep and Circadian Neuroscience Institute, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.; Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK.; Department of Neuroophthalmology, Institute of Ophthalmology, London, UK.
Peirson SN; The Nuffield Laboratory of Ophthalmology, Sleep and Circadian Neuroscience Institute, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.
Hughes S; The Nuffield Laboratory of Ophthalmology, Sleep and Circadian Neuroscience Institute, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.
Hankins MW; The Nuffield Laboratory of Ophthalmology, Sleep and Circadian Neuroscience Institute, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK. .
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Źródło :
Cellular and molecular life sciences : CMLS [Cell Mol Life Sci] 2021 Feb; Vol. 78 (4), pp. 1597-1613. Date of Electronic Publication: 2020 Jul 29.
Typ publikacji :
Journal Article
MeSH Terms :
Genetic Therapy*
Neurons/*metabolism
Retina/*metabolism
Retinal Degeneration/*therapy
Animals ; Channelrhodopsins/genetics ; Channelrhodopsins/therapeutic use ; Clinical Trials as Topic ; Gene Transfer Techniques/trends ; Genetic Vectors/therapeutic use ; Humans ; Light ; Mice ; Neurons/pathology ; Optogenetics ; Retina/pathology ; Retinal Degeneration/genetics ; Retinal Degeneration/pathology
Czasopismo naukowe
Tytuł :
Pluripotent stem cell-based gene therapy approach: human de novo synthesized chromosomes.
Autorzy :
Sinenko SA; Institute of Cytology, Russian Academy of Sciences, 4 Tikhoretsky Ave, St-Petersburg, 194064, Russia. .
Ponomartsev SV; Institute of Cytology, Russian Academy of Sciences, 4 Tikhoretsky Ave, St-Petersburg, 194064, Russia.
Tomilin AN; Institute of Cytology, Russian Academy of Sciences, 4 Tikhoretsky Ave, St-Petersburg, 194064, Russia. .; Institute of Translational Biomedicine, St-Petersburg State University, 7-9, Universitetskaya Emb, St-Petersburg, 199034, Russia. .
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Źródło :
Cellular and molecular life sciences : CMLS [Cell Mol Life Sci] 2021 Feb; Vol. 78 (4), pp. 1207-1220. Date of Electronic Publication: 2020 Oct 03.
Typ publikacji :
Journal Article; Review
MeSH Terms :
Genetic Therapy*
Stem Cell Transplantation*
Chromosomes, Artificial, Human/*genetics
Induced Pluripotent Stem Cells/*transplantation
Gene Transfer Techniques ; Genetic Vectors/genetics ; Genetic Vectors/therapeutic use ; Humans
Czasopismo naukowe
Tytuł :
Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease.
Autorzy :
Esrick EB; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Lehmann LE; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Biffi A; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Achebe M; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Brendel C; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Ciuculescu MF; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Daley H; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
MacKinnon B; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Morris E; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Federico A; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Abriss D; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Boardman K; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Khelladi R; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Shaw K; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Negre H; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Negre O; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Nikiforow S; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Ritz J; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Pai SY; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
London WB; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Dansereau C; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Heeney MM; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Armant M; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Manis JP; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Williams DA; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O'Reilly Families Cell Manipulation Core Facility, Dana-Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) - all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
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Źródło :
The New England journal of medicine [N Engl J Med] 2021 Jan 21; Vol. 384 (3), pp. 205-215. Date of Electronic Publication: 2020 Dec 05.
Typ publikacji :
Clinical Trial; Journal Article; Research Support, N.I.H., Extramural
MeSH Terms :
Genetic Therapy*
RNA Interference*
Anemia, Sickle Cell/*therapy
Fetal Hemoglobin/*biosynthesis
Repressor Proteins/*genetics
gamma-Globins/*metabolism
Adolescent ; Adult ; Anemia, Sickle Cell/genetics ; Child ; Down-Regulation ; Female ; Fetal Hemoglobin/genetics ; Gene Knockdown Techniques ; Genetic Vectors ; Humans ; Male ; Pilot Projects ; RNA, Small Interfering ; Repressor Proteins/metabolism ; Transplantation, Autologous ; Young Adult ; gamma-Globins/genetics
Czasopismo naukowe
Tytuł :
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
Autorzy :
Frangoul H; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Altshuler D; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Cappellini MD; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Chen YS; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Domm J; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Eustace BK; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Foell J; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
de la Fuente J; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Grupp S; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Handgretinger R; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Ho TW; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Kattamis A; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Kernytsky A; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Lekstrom-Himes J; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Li AM; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Locatelli F; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Mapara MY; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
de Montalembert M; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Rondelli D; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Sharma A; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Sheth S; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Soni S; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Steinberg MH; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Wall D; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Yen A; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Corbacioglu S; From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) - both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) - both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children-University of Toronto, Toronto (D.W.) - both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
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Źródło :
The New England journal of medicine [N Engl J Med] 2021 Jan 21; Vol. 384 (3), pp. 252-260. Date of Electronic Publication: 2020 Dec 05.
Typ publikacji :
Clinical Trial; Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms :
CRISPR-Cas Systems*
Genetic Therapy*
Anemia, Sickle Cell/*therapy
Fetal Hemoglobin/*biosynthesis
Gene Editing/*methods
Repressor Proteins/*genetics
beta-Thalassemia/*therapy
Adult ; Anemia, Sickle Cell/genetics ; Female ; Fetal Hemoglobin/genetics ; Humans ; Repressor Proteins/metabolism ; Young Adult ; beta-Thalassemia/genetics
Czasopismo naukowe
Tytuł :
Gene Therapy for Drug-Resistant Glioblastoma via Lipid-Polymer Hybrid Nanoparticles Combined with Focused Ultrasound.
Autorzy :
Yang Q; Department of Neurosurgery, The Second Affiliated Hospital of Chongqing Medical University, Chongqing 400010, People's Republic of China.
Zhou Y; Department of Neurosurgery, The Second Affiliated Hospital of Chongqing Medical University, Chongqing 400010, People's Republic of China.
Chen J; Department of Neurosurgery, The Second Affiliated Hospital of Chongqing Medical University, Chongqing 400010, People's Republic of China.
Huang N; Department of Neurosurgery, The Second Affiliated Hospital of Chongqing Medical University, Chongqing 400010, People's Republic of China.
Wang Z; Institute of Ultrasound Imaging, The Second Affiliated Hospital of Chongqing Medical University, Chongqing 400010, People's Republic of China.
Cheng Y; Department of Neurosurgery, The Second Affiliated Hospital of Chongqing Medical University, Chongqing 400010, People's Republic of China.
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Źródło :
International journal of nanomedicine [Int J Nanomedicine] 2021 Jan 08; Vol. 16, pp. 185-199. Date of Electronic Publication: 2021 Jan 08 (Print Publication: 2021).
Typ publikacji :
Journal Article
MeSH Terms :
Drug Resistance, Neoplasm*
Genetic Therapy*
High-Intensity Focused Ultrasound Ablation*
Brain Neoplasms/*therapy
Glioblastoma/*therapy
Lipids/*chemistry
Nanoparticles/*chemistry
Polymers/*chemistry
Animals ; Blood-Brain Barrier/drug effects ; Brain Neoplasms/drug therapy ; Cell Line, Tumor ; Deoxyribonuclease I/metabolism ; Glioblastoma/drug therapy ; Humans ; Mice, Inbred NOD ; Mice, SCID ; Microbubbles ; Nanoparticles/ultrastructure ; Peptides, Cyclic/chemistry ; Plasmids/genetics ; Temozolomide/pharmacology ; Temozolomide/therapeutic use ; Tissue Distribution ; Transfection
Czasopismo naukowe
Tytuł :
Epilepsy in 2020-a new dawn.
Autorzy :
Cross JH; UCL National Institute of Health Research BRC Great Ormond Street Institute of Child Health, London WC1N 1EH, UK. Electronic address: .
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Źródło :
The Lancet. Neurology [Lancet Neurol] 2021 Jan; Vol. 20 (1), pp. 8-10.
Typ publikacji :
Journal Article
MeSH Terms :
Genetic Therapy*
Epilepsy/*therapy
Fenfluramine/*pharmacology
Serotonin Uptake Inhibitors/*pharmacology
Animals ; Clinical Trials as Topic ; Databases as Topic ; Epilepsies, Myoclonic/therapy ; Epilepsy/drug therapy ; Epilepsy/genetics ; Epilepsy/pathology ; Humans ; NAV1.1 Voltage-Gated Sodium Channel ; Neuroimaging
Czasopismo naukowe
Tytuł :
CRISPR technology: The engine that drives cancer therapy.
Autorzy :
Zhang M; Guangdong Key Laboratory for Research and Development of Natural Drugs, The Marine Biomedical Research Institute, Guangdong Medical University, Zhanjiang, China; Southern Marine Science and Engineering Guangdong Laboratory (Zhanjiang), Zhanjiang, China.
Eshraghian EA; Department of Family Medicine and Public Health, School of Medicine, University of California San Diego, CA 92093, USA.
Jammal OA; Department of Family Medicine and Public Health, School of Medicine, University of California San Diego, CA 92093, USA.
Zhang Z; Biomedical Engineering Research Center, Kunming Medical University, Kunming, China. Electronic address: .
Zhu X; Guangdong Key Laboratory for Research and Development of Natural Drugs, The Marine Biomedical Research Institute, Guangdong Medical University, Zhanjiang, China; Southern Marine Science and Engineering Guangdong Laboratory (Zhanjiang), Zhanjiang, China; The Marine Biomedical Research Institute of Guangdong Zhanjiang, Zhanjiang, China; The Key Lab of Zhanjiang for R&D Marine Microbial Resources in the Beibu Gulf Rim, Guangdong Medical University, Zhanjiang, China. Electronic address: .
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Źródło :
Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie [Biomed Pharmacother] 2021 Jan; Vol. 133, pp. 111007. Date of Electronic Publication: 2020 Nov 20.
Typ publikacji :
Journal Article; Review
MeSH Terms :
CRISPR-Cas Systems*
Clustered Regularly Interspaced Short Palindromic Repeats*
Gene Editing*
Genetic Therapy*/adverse effects
Immunotherapy*/adverse effects
Molecular Targeted Therapy*/adverse effects
CRISPR-Associated Proteins/*genetics
Neoplasms/*therapy
Animals ; CRISPR-Associated Proteins/metabolism ; Diffusion of Innovation ; Gene Expression Regulation, Neoplastic ; Gene Transfer Techniques ; Humans ; Molecular Diagnostic Techniques ; Neoplasms/genetics ; Neoplasms/immunology ; Neoplasms/metabolism ; Predictive Value of Tests
Czasopismo naukowe
Tytuł :
Treatment of one eye affects both.
Autorzy :
Stower H; Nature Medicine, . .
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Źródło :
Nature medicine [Nat Med] 2021 Jan; Vol. 27 (1), pp. 21.
Typ publikacji :
Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial
MeSH Terms :
Genetic Therapy*
Optic Atrophy, Hereditary, Leber/*therapy
Administration, Ophthalmic ; Humans ; Optic Atrophy, Hereditary, Leber/physiopathology ; Vision, Ocular
Czasopismo naukowe
Tytuł :
APOE and Alzheimer's disease: advances in genetics, pathophysiology, and therapeutic approaches.
Autorzy :
Serrano-Pozo A; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA; Massachusetts Alzheimer's Disease Research Center, Charlestown, MA, USA; Harvard Medical School, Boston, MA, USA.
Das S; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA; Massachusetts Alzheimer's Disease Research Center, Charlestown, MA, USA; Harvard Medical School, Boston, MA, USA.
Hyman BT; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA; Massachusetts Alzheimer's Disease Research Center, Charlestown, MA, USA; Harvard Medical School, Boston, MA, USA. Electronic address: .
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Źródło :
The Lancet. Neurology [Lancet Neurol] 2021 Jan; Vol. 20 (1), pp. 68-80.
Typ publikacji :
Journal Article; Review
MeSH Terms :
Alzheimer Disease*/drug therapy
Alzheimer Disease*/genetics
Alzheimer Disease*/metabolism
Alzheimer Disease*/pathology
Apolipoproteins E*/drug effects
Apolipoproteins E*/genetics
Apolipoproteins E*/metabolism
Genetic Therapy*
Animals ; Humans
Czasopismo naukowe
Tytuł :
An update on ACE2 amplification and its therapeutic potential.
Autorzy :
Marquez A; Feinberg Medical School, Northwestern University, Chicago, IL, USA.; Department of Medicine Division of Nephrology and Hypertension, Chicago, IL, USA.
Wysocki J; Feinberg Medical School, Northwestern University, Chicago, IL, USA.; Department of Medicine Division of Nephrology and Hypertension, Chicago, IL, USA.
Pandit J; Feinberg Medical School, Northwestern University, Chicago, IL, USA.; Department of Medicine Division of Nephrology and Hypertension, Chicago, IL, USA.
Batlle D; Feinberg Medical School, Northwestern University, Chicago, IL, USA.; Department of Medicine Division of Nephrology and Hypertension, Chicago, IL, USA.
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Źródło :
Acta physiologica (Oxford, England) [Acta Physiol (Oxf)] 2021 Jan; Vol. 231 (1), pp. e13513. Date of Electronic Publication: 2020 Jun 17.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Review
MeSH Terms :
Angiotensin-Converting Enzyme 2*/genetics
Angiotensin-Converting Enzyme 2*/metabolism
Angiotensin-Converting Enzyme 2*/therapeutic use
Genetic Therapy*
Receptors, Virus*/genetics
Receptors, Virus*/metabolism
Receptors, Virus*/therapeutic use
Antiviral Agents/*therapeutic use
COVID-19/*drug therapy
COVID-19/*therapy
SARS-CoV-2/*pathogenicity
Virus Internalization/*drug effects
Animals ; COVID-19/enzymology ; COVID-19/genetics ; COVID-19/virology ; Enzyme Activation ; Enzyme Activators/therapeutic use ; Gene Amplification ; Host-Pathogen Interactions ; Humans ; Recombinant Proteins/therapeutic use
SCR Protocol :
COVID-19 drug treatment
Czasopismo naukowe
Tytuł :
Development of New Oncolytic Virotherapy Targeting Breast Cancer Using Coxsackievirus B3.
Autorzy :
Sagara M; Laboratory of ALA Advanced Medical Research, Institute for Quantitative Biosciences, The University of Tokyo, Tokyo, Japan.
Miyamoto S; Laboratory of ALA Advanced Medical Research, Institute for Quantitative Biosciences, The University of Tokyo, Tokyo, Japan.
Itoh S; Laboratory of ALA Advanced Medical Research, Institute for Quantitative Biosciences, The University of Tokyo, Tokyo, Japan.
Soda Y; Laboratory of ALA Advanced Medical Research, Institute for Quantitative Biosciences, The University of Tokyo, Tokyo, Japan.
Tani K; Laboratory of ALA Advanced Medical Research, Institute for Quantitative Biosciences, The University of Tokyo, Tokyo, Japan .
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Źródło :
Anticancer research [Anticancer Res] 2021 Jan; Vol. 41 (1), pp. 81-89.
Typ publikacji :
Journal Article
MeSH Terms :
Genetic Therapy*/methods
Oncolytic Virotherapy*/methods
Breast Neoplasms/*genetics
Enterovirus B, Human/*genetics
Genetic Vectors/*genetics
Oncolytic Viruses/*genetics
Animals ; Breast Neoplasms/pathology ; Breast Neoplasms/therapy ; Cell Line, Tumor ; Cells, Cultured ; Cytopathogenic Effect, Viral ; Disease Models, Animal ; Female ; Flow Cytometry ; Gene Expression ; Genetic Vectors/administration & dosage ; Genetic Vectors/adverse effects ; Humans ; Immunohistochemistry ; Mice ; Treatment Outcome ; Xenograft Model Antitumor Assays
Czasopismo naukowe
Tytuł :
Patient and public perspectives on cell and gene therapies: a systematic review.
Autorzy :
Aiyegbusi OL; Centre for Patient Reported Outcomes Research, Institute of Applied Health Research, University of Birmingham, Birmingham, UK. .; National Institute for Health Research Birmingham Biomedical Research Centre, University of Birmingham, Birmingham, UK. .; National Institute for Health Research Applied Research Centre West Midlands, and National Institute for Health Research Surgical Reconstruction and Microbiology Research Centre, University of Birmingham, Birmingham, UK. .; Birmingham Health Partners Centre for Regulatory Science and Innovation, Birmingham, UK. .
Macpherson K; Healthcare Improvement Scotland, Glasgow, Scotland, UK.
Elston L; Health Technology Wales, Cardiff, UK.
Myles S; Health Technology Wales, Cardiff, UK.
Washington J; Health Technology Wales, Cardiff, UK.
Sungum N; Birmingham Health Partners Centre for Regulatory Science and Innovation, Birmingham, UK.; Midlands-Wales Advanced Therapy Treatment Centre, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.
Briggs M; Welsh Blood Service, Velindre University NHS Trust, Cardiff, UK.
Newsome PN; National Institute for Health Research Birmingham Biomedical Research Centre, University of Birmingham, Birmingham, UK.; National Institute for Health Research Applied Research Centre West Midlands, and National Institute for Health Research Surgical Reconstruction and Microbiology Research Centre, University of Birmingham, Birmingham, UK.; Birmingham Health Partners Centre for Regulatory Science and Innovation, Birmingham, UK.; Centre for Liver and Gastrointestinal Research, Institute of Immunology and Immunotherapy, University of Birmingham, Birmingham, UK.; Liver Unit, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.
Calvert MJ; Centre for Patient Reported Outcomes Research, Institute of Applied Health Research, University of Birmingham, Birmingham, UK.; National Institute for Health Research Birmingham Biomedical Research Centre, University of Birmingham, Birmingham, UK.; National Institute for Health Research Applied Research Centre West Midlands, and National Institute for Health Research Surgical Reconstruction and Microbiology Research Centre, University of Birmingham, Birmingham, UK.; Birmingham Health Partners Centre for Regulatory Science and Innovation, Birmingham, UK.
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Źródło :
Nature communications [Nat Commun] 2020 Dec 08; Vol. 11 (1), pp. 6265. Date of Electronic Publication: 2020 Dec 08.
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't; Systematic Review
MeSH Terms :
Public Opinion*
Cell Transplantation/*methods
Delivery of Health Care/*ethics
Genetic Therapy/*methods
Cell Transplantation/adverse effects ; Cell Transplantation/economics ; Cell Transplantation/ethics ; Cost-Benefit Analysis ; Delivery of Health Care/economics ; Genetic Therapy/adverse effects ; Genetic Therapy/economics ; Genetic Therapy/ethics ; Health Education ; Humans ; Patient Education as Topic ; Qualitative Research
Czasopismo naukowe
Tytuł :
[Anti-TNF-α in the treatment of non-infectious uveitis].
Autorzy :
Hoogewoud F; Ophtalmopole Hôpital Cochin; Assistance Publique-Hôpitaux de Paris; Université de Paris, 27 rue du Faubourg Saint-Jacques, 75014 Paris, France - Département d'ophtalmologie de l'université de Lausanne, Hôpital Ophtalmologique Jules-Gonin, Lausanne, Suisse.
Kowalczuk L; Département d'ophtalmologie de l'université de Lausanne, Hôpital Ophtalmologique Jules-Gonin, Lausanne, Suisse - Centre de recherches des Cordeliers; Inserm UMR 1138, Physiopathologie des maladies oculaires : innovations thérapeutiques; Université de Paris, 15 rue de l'École de Médecine, 75006 Paris, France.
Bousquet E; Ophtalmopole Hôpital Cochin; Assistance Publique-Hôpitaux de Paris; Université de Paris, 27 rue du Faubourg Saint-Jacques, 75014 Paris, France - Centre de recherches des Cordeliers; Inserm UMR 1138, Physiopathologie des maladies oculaires : innovations thérapeutiques; Université de Paris, 15 rue de l'École de Médecine, 75006 Paris, France.
Brézin A; Ophtalmopole Hôpital Cochin; Assistance Publique-Hôpitaux de Paris; Université de Paris, 27 rue du Faubourg Saint-Jacques, 75014 Paris, France - Centre de recherches des Cordeliers; Inserm UMR 1138, Physiopathologie des maladies oculaires : innovations thérapeutiques; Université de Paris, 15 rue de l'École de Médecine, 75006 Paris, France.
Touchard E; Eyevensys, SAS, Paris, France.
Buggage R; Eyevensys, SAS, Paris, France.
Bordet T; Eyevensys, SAS, Paris, France.
Behar-Cohen F; Ophtalmopole Hôpital Cochin; Assistance Publique-Hôpitaux de Paris; Université de Paris, 27 rue du Faubourg Saint-Jacques, 75014 Paris, France - Centre de recherches des Cordeliers; Inserm UMR 1138, Physiopathologie des maladies oculaires : innovations thérapeutiques; Université de Paris, 15 rue de l'École de Médecine, 75006 Paris, France.
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Transliterated Title :
Les anti-TNF-α pour le traitement des uvéites non infectieuses.
Źródło :
Medecine sciences : M/S [Med Sci (Paris)] 2020 Oct; Vol. 36 (10), pp. 893-899. Date of Electronic Publication: 2020 Oct 07.
Typ publikacji :
Journal Article; Review
MeSH Terms :
Genetic Therapy*/methods
Genetic Therapy*/trends
Tumor Necrosis Factor-alpha/*immunology
Uveitis/*therapy
Vision Disorders/*prevention & control
Antibodies, Neutralizing/genetics ; Antibodies, Neutralizing/therapeutic use ; Autoimmune Diseases/therapy ; Humans ; Tumor Necrosis Factor-alpha/antagonists & inhibitors
Czasopismo naukowe
Tytuł :
HK022 bacteriophage Integrase mediated RMCE as a potential tool for human gene therapy.
Autorzy :
Elias A; Department of Biochemistry and Molecular Biology, School of Neurobiology, Biochemistry &Biophysics, Tel-Aviv University, Tel-Aviv, Israel.
Kassis H; Department of Biochemistry and Molecular Biology, School of Neurobiology, Biochemistry &Biophysics, Tel-Aviv University, Tel-Aviv, Israel.
Elkader SA; Department of Biochemistry and Molecular Biology, School of Neurobiology, Biochemistry &Biophysics, Tel-Aviv University, Tel-Aviv, Israel.
Gritsenko N; Department of Biochemistry and Molecular Biology, School of Neurobiology, Biochemistry &Biophysics, Tel-Aviv University, Tel-Aviv, Israel.
Nahmad A; Department of Biochemistry and Molecular Biology, School of Neurobiology, Biochemistry &Biophysics, Tel-Aviv University, Tel-Aviv, Israel.
Shir H; Department of Biochemistry and Molecular Biology, School of Neurobiology, Biochemistry &Biophysics, Tel-Aviv University, Tel-Aviv, Israel.
Younis L; Department of Biochemistry and Molecular Biology, School of Neurobiology, Biochemistry &Biophysics, Tel-Aviv University, Tel-Aviv, Israel.
Shannan A; Department of Biochemistry and Molecular Biology, School of Neurobiology, Biochemistry &Biophysics, Tel-Aviv University, Tel-Aviv, Israel.
Aihara H; Department of Biochemistry, Molecular Biology, and Biophysics, University of Minnesota TwinCities, Minneapolis, MN, 55455, USA.
Prag G; Department of Biochemistry and Molecular Biology, School of Neurobiology, Biochemistry &Biophysics, Tel-Aviv University, Tel-Aviv, Israel.
Yagil E; Department of Biochemistry and Molecular Biology, School of Neurobiology, Biochemistry &Biophysics, Tel-Aviv University, Tel-Aviv, Israel.
Kolot M; Department of Biochemistry and Molecular Biology, School of Neurobiology, Biochemistry &Biophysics, Tel-Aviv University, Tel-Aviv, Israel.
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Źródło :
Nucleic acids research [Nucleic Acids Res] 2020 Dec 16; Vol. 48 (22), pp. 12804-12816.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
MeSH Terms :
Genetic Therapy*
Bacteriophage HK022/*genetics
Integrases/*genetics
Recombination, Genetic/*genetics
Bacteriophage HK022/enzymology ; DNA Nucleotidyltransferases/genetics ; Genome, Human/genetics ; Humans
Czasopismo naukowe
Tytuł :
Comparative intra-articular gene transfer of seven adeno-associated virus serotypes reveals that AAV2 mediates the most efficient transduction to mouse arthritic chondrocytes.
Autorzy :
Chen Q; The First Affiliated Hospital of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.; The First Clinical Medical College of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.
Luo H; Department of Pharmacy, The Second Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou, China.
Zhou C; The First Affiliated Hospital of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.; The First Clinical Medical College of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.
Yu H; The First Affiliated Hospital of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.; The First Clinical Medical College of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.
Yao S; The First Affiliated Hospital of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.; The First Clinical Medical College of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.
Fu F; The First Affiliated Hospital of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.; The First Clinical Medical College of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.
Seeley R; Translational Research Program in Pediatric Orthopedics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States of America.
Ji X; Translational Research Program in Pediatric Orthopedics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States of America.
Yang Y; Longhua Hospital Affiliated to Shanghai University of Traditional Chinese Medicine, Shanghai, China.
Chen P; The First Affiliated Hospital of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.; The First Clinical Medical College of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.
Jin H; The First Affiliated Hospital of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.; The First Clinical Medical College of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.
Tong P; The First Affiliated Hospital of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.; The First Clinical Medical College of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.
Chen D; Research Center for Human Tissues and Organs Degeneration, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen, China.
Wu C; The First Affiliated Hospital of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.; The First Clinical Medical College of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.
Du W; Research Institute of Orthopedics, the Affiliated JiangNan Hospital of Zhejiang Chinese Medical University, Hangzhou, China.
Ruan H; The First Affiliated Hospital of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.; The First Clinical Medical College of Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.; Longhua Hospital Affiliated to Shanghai University of Traditional Chinese Medicine, Shanghai, China.
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Źródło :
PloS one [PLoS One] 2020 Dec 15; Vol. 15 (12), pp. e0243359. Date of Electronic Publication: 2020 Dec 15 (Print Publication: 2020).
Typ publikacji :
Comparative Study; Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms :
Arthritis, Experimental*/genetics
Arthritis, Experimental*/metabolism
Arthritis, Experimental*/pathology
Arthritis, Experimental*/therapy
Genetic Therapy*
Genetic Vectors*
Parvovirinae*
Cartilage, Articular/*metabolism
Chondrocytes/*metabolism
Knee Joint/*metabolism
ADAMTS4 Protein/biosynthesis ; ADAMTS4 Protein/genetics ; Animals ; Cartilage, Articular/pathology ; Chondrocytes/pathology ; Collagen Type II/biosynthesis ; Collagen Type II/genetics ; Knee Joint/pathology ; Male ; Matrix Metalloproteinases, Secreted/biosynthesis ; Matrix Metalloproteinases, Secreted/genetics ; Mice ; Transduction, Genetic
SCR Organism :
Adeno-associated virus-2
Czasopismo naukowe

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