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Tytuł :
Efficient CRISPR/Cas9 nickase-mediated genome editing in an in vitro model of mucopolysaccharidosis IVA.
Autorzy :
Leal AF; Institute for the Study of Inborn Errors of Metabolism, Faculty of Science, Pontificia Universidad Javeriana, Bogotá D.C., 110231, Colombia.
Alméciga-Díaz CJ; Institute for the Study of Inborn Errors of Metabolism, Faculty of Science, Pontificia Universidad Javeriana, Bogotá D.C., 110231, Colombia. .
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Źródło :
Gene therapy [Gene Ther] 2022 May 18. Date of Electronic Publication: 2022 May 18.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Optimized expression of alternative oxidase.
Autorzy :
Neuzil J; School of Pharmacy and Medical Science, Griffith University, Southport, QLD, Australia. .; Institute of Biotechnology, Czech Academy of Sciences, Prague-West, Czech Republic. .
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Źródło :
Gene therapy [Gene Ther] 2022 May 17. Date of Electronic Publication: 2022 May 17.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
CRISPR/Cas9-based genome editing for the modification of multiple duplications that cause Duchenne muscular dystrophy.
Autorzy :
Wang DN; Department of Neurology, The First Affiliated Hospital of Xiamen University, School of Medicine, Xiamen University, Xiamen, China.
Wang ZQ; Department of Neurology and Institute of Neurology of First Affiliated Hospital, Institute of Neuroscience, and Fujian Key Laboratory of Molecular Neurology, Fujian Medical University, Fuzhou, China.
Jin M; Department of Neurology and Institute of Neurology of First Affiliated Hospital, Institute of Neuroscience, and Fujian Key Laboratory of Molecular Neurology, Fujian Medical University, Fuzhou, China.
Lin MT; Department of Neurology and Institute of Neurology of First Affiliated Hospital, Institute of Neuroscience, and Fujian Key Laboratory of Molecular Neurology, Fujian Medical University, Fuzhou, China.
Wang N; Department of Neurology and Institute of Neurology of First Affiliated Hospital, Institute of Neuroscience, and Fujian Key Laboratory of Molecular Neurology, Fujian Medical University, Fuzhou, China. .
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Źródło :
Gene therapy [Gene Ther] 2022 May 09. Date of Electronic Publication: 2022 May 09.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
HIV- 1 lentivirus tethering to the genome is associated with transcription factor binding sites found in genes that favour virus survival.
Autorzy :
Suleman S; Department of Life Sciences, College of Health, Medicine & Life Sciences, Brunel University London, Uxbridge, UK.; Testavec Ltd, Queensgate House, Maidenhead, UK.
Payne A; Testavec Ltd, Queensgate House, Maidenhead, UK.; Department of Computer Science, College of Engineering Design and Physical Sciences, Brunel University London, Uxbridge, UK.
Bowden J; Department of Life Sciences, College of Health, Medicine & Life Sciences, Brunel University London, Uxbridge, UK.
Haque SA; Department of Life Sciences, College of Health, Medicine & Life Sciences, Brunel University London, Uxbridge, UK.
Zahn M; Genewerk GmbH, Heidelberg, Germany.; University Heidelberg, Medical Faculty, Heidelberg, Germany.
Fawaz S; Department of Life Sciences, College of Health, Medicine & Life Sciences, Brunel University London, Uxbridge, UK.
Khalifa MS; Department of Life Sciences, College of Health, Medicine & Life Sciences, Brunel University London, Uxbridge, UK.
Jobling S; Testavec Ltd, Queensgate House, Maidenhead, UK.; Institute of Environment, Health and Societies, College of Business, Arts and Social Sciences, Brunel University London, Uxbridge, UK.
Hay D; Centre for Regenerative Medicine, The University of Edinburgh, Edinburgh, UK.
Franco M; Genewerk GmbH, Heidelberg, Germany.
Fronza R; Genewerk GmbH, Heidelberg, Germany.
Wang W; Genewerk GmbH, Heidelberg, Germany.
Strobel-Freidekind O; Genewerk GmbH, Heidelberg, Germany.
Deichmann A; Genewerk GmbH, Heidelberg, Germany.
Takeuchi Y; Division of Infection and Immunity, University College London, London, UK.; Division of Advanced Therapies, National Institute for Biological Standards and Control, Potters Bar, UK.
Waddington SN; Gene Transfer Technology, EGA Institute for Women's Health, University College London, London, UK.; MRC Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witswatersrand, Johannesburg, South Africa.
Gil-Farina I; Genewerk GmbH, Heidelberg, Germany.
Schmidt M; Genewerk GmbH, Heidelberg, Germany.; Department of Translational Oncology, NCT and DKFZ, Heidelberg, Germany.
Themis M; Department of Life Sciences, College of Health, Medicine & Life Sciences, Brunel University London, Uxbridge, UK. .; Division of Ecology and Evolution, Department of Life Sciences, Imperial College London, London, UK. .
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Źródło :
Gene therapy [Gene Ther] 2022 May 05. Date of Electronic Publication: 2022 May 05.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec -routine care evidence.
Autorzy :
Aharoni S; Institute of Pediatric Neurology, Schneider Children's Medical Center of Israel, Petach Tikva, Israel. .; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel. .
Bistritzer J; Institute of Pediatric Neurology, Schneider Children's Medical Center of Israel, Petach Tikva, Israel.; Pediatric Neurology Unit, Soroka University Medical Center, Ben Gurion University, Beer Sheva, Israel.
Levine H; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.; Pulmonary Institute, Schneider Children's Medical Center, Petach Tikva, Israel.
Sagi L; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.; Pediatric Neurology Institute, Dana-Dwek Children's Hospital, Tel Aviv Medical Center, Tel Aviv, Israel.
Fattal-Valevski A; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.; Pediatric Neurology Institute, Dana-Dwek Children's Hospital, Tel Aviv Medical Center, Tel Aviv, Israel.
Ginzberg M; Pediatric Neurology Unit, Wolfson Medical Center, Holon, Israel.
Noyman I; Pediatric Neurology Unit, Soroka University Medical Center, Ben Gurion University, Beer Sheva, Israel.
Cohen R; Institute of Pediatric Neurology, Schneider Children's Medical Center of Israel, Petach Tikva, Israel.; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
Nevo Y; Institute of Pediatric Neurology, Schneider Children's Medical Center of Israel, Petach Tikva, Israel.; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
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Źródło :
Gene therapy [Gene Ther] 2022 Apr 27. Date of Electronic Publication: 2022 Apr 27.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy.
Autorzy :
Reilly A; Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, Canada.; Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, Canada.; Centre for Neuromuscular Disease, University of Ottawa, Ottawa, Canada.
Deguise MO; Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, Canada.; Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, Canada.; Centre for Neuromuscular Disease, University of Ottawa, Ottawa, Canada.
Beauvais A; Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, Canada.
Yaworski R; Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, Canada.
Thebault S; Division of Neurology, Department of Medicine, The Ottawa Hospital, Ottawa, Canada.; Department of Medicine, University of Ottawa, Ottawa, Canada.
Tessier DR; Department of Physics, University of Ottawa, Ottawa, Canada.
Tabard-Cossa V; Department of Physics, University of Ottawa, Ottawa, Canada.
Hensel N; Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, Canada.; Centre for Neuromuscular Disease, University of Ottawa, Ottawa, Canada.
Schneider BL; Brain Mind Institute, Ecole Polytechnique Fédérale de Lausanne, Lausanne, Switzerland.; Bertarelli Platform for Gene Therapy, Ecole Polytechnique Fédérale de Lausanne, Geneva, Switzerland.
Kothary R; Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, Canada. .; Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, Canada. .; Centre for Neuromuscular Disease, University of Ottawa, Ottawa, Canada. .; Department of Medicine, University of Ottawa, Ottawa, Canada. .
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Źródło :
Gene therapy [Gene Ther] 2022 Apr 25. Date of Electronic Publication: 2022 Apr 25.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Micro-dystrophin gene constructs for repairing heart and muscle function in rats: the smaller is enough?
Autorzy :
Szabo PL; Ludwig Boltzmann Institute for Cardiovascular Research at the Center for Biomedical Research, Medical University of Vienna, Vienna, Austria.
Kiss A; Ludwig Boltzmann Institute for Cardiovascular Research at the Center for Biomedical Research, Medical University of Vienna, Vienna, Austria. .
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Źródło :
Gene therapy [Gene Ther] 2022 Apr 22. Date of Electronic Publication: 2022 Apr 22.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Exosomal PD-L1 confers chemoresistance and promotes tumorigenic properties in esophageal cancer cells via upregulating STAT3/miR-21.
Autorzy :
Wang H; School of Basic Medical Sciences, Henan University of Science & Technology, Luoyang, China.
Qi Y; Henan Key Laboratory of Microbiome and Esophageal Cancer Prevention and Treatment, The First Affiliated Hospital School of Clinical Medicine of Henan University of Science and Technology, Luoyang, China.
Lan Z; Henan Key Laboratory of Microbiome and Esophageal Cancer Prevention and Treatment, The First Affiliated Hospital School of Clinical Medicine of Henan University of Science and Technology, Luoyang, China.
Liu Q; Key Laboratory of Cell Behavior, Medical College of Xuchang University, Xuchang, China.
Xu J; School of Basic Medical Sciences, Henan University of Science & Technology, Luoyang, China.
Zhu M; School of Basic Medical Sciences, Henan University of Science & Technology, Luoyang, China.
Yang T; School of Basic Medical Sciences, Henan University of Science & Technology, Luoyang, China.
Shi R; School of Basic Medical Sciences, Henan University of Science & Technology, Luoyang, China.
Gao S; Henan Key Laboratory of Microbiome and Esophageal Cancer Prevention and Treatment, The First Affiliated Hospital School of Clinical Medicine of Henan University of Science and Technology, Luoyang, China. .
Liang G; School of Basic Medical Sciences, Henan University of Science & Technology, Luoyang, China. .
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Źródło :
Gene therapy [Gene Ther] 2022 Apr 19. Date of Electronic Publication: 2022 Apr 19.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Correction: Maximizing lentiviral vector gene transfer in the CNS.
Autorzy :
Humbel M; Lausanne University Hospital (CHUV) and University of Lausanne, Department of Clinical Neurosciences (DNC), Laboratory of Neurotherapies and NeuroModulation, Lausanne, Switzerland.; Lausanne University Hospital (CHUV) and University of Lausanne, Neuroscience Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne, Switzerland.
Ramosaj M; Lausanne University Hospital (CHUV) and University of Lausanne, Department of Clinical Neurosciences (DNC), Laboratory of Neurotherapies and NeuroModulation, Lausanne, Switzerland.; Lausanne University Hospital (CHUV) and University of Lausanne, Neuroscience Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne, Switzerland.
Zimmer V; Lausanne University Hospital (CHUV) and University of Lausanne, Department of Clinical Neurosciences (DNC), Laboratory of Neurotherapies and NeuroModulation, Lausanne, Switzerland.; Lausanne University Hospital (CHUV) and University of Lausanne, Neuroscience Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne, Switzerland.
Regio S; Lausanne University Hospital (CHUV) and University of Lausanne, Department of Clinical Neurosciences (DNC), Laboratory of Neurotherapies and NeuroModulation, Lausanne, Switzerland.; Lausanne University Hospital (CHUV) and University of Lausanne, Neuroscience Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne, Switzerland.
Aeby L; Lausanne University Hospital (CHUV) and University of Lausanne, Department of Clinical Neurosciences (DNC), Laboratory of Neurotherapies and NeuroModulation, Lausanne, Switzerland.; Lausanne University Hospital (CHUV) and University of Lausanne, Neuroscience Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne, Switzerland.
Moser S; Lausanne University Hospital (CHUV) and University of Lausanne, Department of Clinical Neurosciences (DNC), Laboratory of Neurotherapies and NeuroModulation, Lausanne, Switzerland.; Lausanne University Hospital (CHUV) and University of Lausanne, Neuroscience Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne, Switzerland.
Boizot A; Lausanne University Hospital (CHUV) and University of Lausanne, Department of Clinical Neurosciences (DNC), Laboratory of Neurotherapies and NeuroModulation, Lausanne, Switzerland.; Lausanne University Hospital (CHUV) and University of Lausanne, Neuroscience Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne, Switzerland.
Sipion M; Lausanne University Hospital (CHUV) and University of Lausanne, Department of Clinical Neurosciences (DNC), Laboratory of Neurotherapies and NeuroModulation, Lausanne, Switzerland.; Lausanne University Hospital (CHUV) and University of Lausanne, Neuroscience Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne, Switzerland.
Rey M; Lausanne University Hospital (CHUV) and University of Lausanne, Department of Clinical Neurosciences (DNC), Laboratory of Neurotherapies and NeuroModulation, Lausanne, Switzerland.; Lausanne University Hospital (CHUV) and University of Lausanne, Neuroscience Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne, Switzerland.
Déglon N; Lausanne University Hospital (CHUV) and University of Lausanne, Department of Clinical Neurosciences (DNC), Laboratory of Neurotherapies and NeuroModulation, Lausanne, Switzerland. .; Lausanne University Hospital (CHUV) and University of Lausanne, Neuroscience Research Center (CRN), Laboratory of Cellular and Molecular Neurotherapies (LCMN), Lausanne, Switzerland. .; Laboratory of Neurotherapies and Neuromodulation, Neuroscience reserach Center (CRN), Lausanne Univeristy Hospital (CHUV), Avenue de Beaumont, Pavillon 3, Lausanne, Switzerland. .
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Źródło :
Gene therapy [Gene Ther] 2022 Apr 12. Date of Electronic Publication: 2022 Apr 12.
Typ publikacji :
Published Erratum
Tytuł :
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene.
Autorzy :
Liu Y; Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, FL, USA.
Eastwood JD; Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, FL, USA.
Alba DE; Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, FL, USA.
Velmurugan S; Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, FL, USA.
Sun N; Department of Biostatistics, Robert Stempel College of Public Health & Social Work, Florida International University, Miami, FL, USA.
Porciatti V; Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, FL, USA.
Lee RK; Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, FL, USA. .
Hauswirth WW; Department of Ophthalmology, College of Medicine, University of Florida, Gainesville, FL, USA.
Guy J; Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, FL, USA.
Yu H; Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, FL, USA. .
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Źródło :
Gene therapy [Gene Ther] 2022 Apr 06. Date of Electronic Publication: 2022 Apr 06.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Control of gene doping in human and horse sports.
Autorzy :
Tozaki T; Genetic Analysis Department, Laboratory of Racing Chemistry, Tochigi, 320-0851, Japan. .
Hamilton NA; Equine Genetics Research Centre, Racing Australia, Flemington, NSW, 2337, Australia.
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Źródło :
Gene therapy [Gene Ther] 2022 Apr; Vol. 29 (3-4), pp. 107-112. Date of Electronic Publication: 2021 Jun 07.
Typ publikacji :
Journal Article
MeSH Terms :
Doping in Sports*
Erythropoietin*/genetics
Animals ; Horses/genetics ; Humans ; Transgenes
Czasopismo naukowe
Tytuł :
XIAP gene therapy effects on retinal ganglion cell structure and function in a mouse model of glaucoma.
Autorzy :
Visuvanathan S; Department of Biochemistry, Microbiology and Immunology, University of Ottawa, Ottawa, ON, Canada.
Baker AN; Neuroscience, Ottawa Hospital Research Institute, Ottawa, ON, Canada.
Lagali PS; Neuroscience, Ottawa Hospital Research Institute, Ottawa, ON, Canada.
Coupland SG; Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, ON, Canada.; Regenerative Medicine, Ottawa Hospital Research Institute, Ottawa, ON, Canada.; Department of Ophthalmology, University of Ottawa, Ottawa, ON, Canada.
Miller G; Regenerative Medicine, Ottawa Hospital Research Institute, Ottawa, ON, Canada.; Department of Ophthalmology, University of Ottawa, Ottawa, ON, Canada.
Hauswirth WW; Department of Ophthalmology, University of Florida College of Medicine, Gainesville, FL, USA.
Tsilfidis C; Neuroscience, Ottawa Hospital Research Institute, Ottawa, ON, Canada. .; Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, ON, Canada. .; Department of Ophthalmology, University of Ottawa, Ottawa, ON, Canada. .
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Źródło :
Gene therapy [Gene Ther] 2022 Apr; Vol. 29 (3-4), pp. 147-156. Date of Electronic Publication: 2021 Aug 06.
Typ publikacji :
Journal Article
MeSH Terms :
Glaucoma*/genetics
Glaucoma*/therapy
Neurodegenerative Diseases*
Animals ; Axons ; Disease Models, Animal ; Genetic Therapy ; Intraocular Pressure ; Mice ; Retinal Ganglion Cells/metabolism
Czasopismo naukowe
Tytuł :
Effects of oncolytic viruses and viral vectors on immunity in glioblastoma.
Autorzy :
Liu P; Departments of Neurosurgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China.
Wang Y; Departments of Neurosurgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China.
Wang Y; Departments of Neurosurgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China.
Kong Z; Departments of Neurosurgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China.
Chen W; Departments of Neurosurgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China.
Li J; Departments of Neurosurgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China.
Chen W; Departments of Neurosurgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China.
Tong Y; Departments of Neurosurgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China.
Ma W; Departments of Neurosurgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China. .
Wang Y; Departments of Neurosurgery, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China. .
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Źródło :
Gene therapy [Gene Ther] 2022 Apr; Vol. 29 (3-4), pp. 115-126. Date of Electronic Publication: 2020 Nov 15.
Typ publikacji :
Journal Article; Review
MeSH Terms :
Brain Neoplasms*/therapy
Glioblastoma*/genetics
Glioblastoma*/therapy
Oncolytic Virotherapy*
Oncolytic Viruses*/genetics
Humans ; Tumor Microenvironment
Czasopismo naukowe
Tytuł :
Upper motor neurons are a target for gene therapy and UCHL1 is necessary and sufficient to improve cellular integrity of diseased upper motor neurons.
Autorzy :
Genç B; Davee Department of Neurology and Clinical Neurological Sciences, Northwestern University, Feinberg School of Medicine, Chicago, IL, 60611, USA.
Jara JH; Davee Department of Neurology and Clinical Neurological Sciences, Northwestern University, Feinberg School of Medicine, Chicago, IL, 60611, USA.
Sanchez SS; Davee Department of Neurology and Clinical Neurological Sciences, Northwestern University, Feinberg School of Medicine, Chicago, IL, 60611, USA.
Lagrimas AKB; Davee Department of Neurology and Clinical Neurological Sciences, Northwestern University, Feinberg School of Medicine, Chicago, IL, 60611, USA.
Gözütok Ö; Davee Department of Neurology and Clinical Neurological Sciences, Northwestern University, Feinberg School of Medicine, Chicago, IL, 60611, USA.
Koçak N; Davee Department of Neurology and Clinical Neurological Sciences, Northwestern University, Feinberg School of Medicine, Chicago, IL, 60611, USA.
Zhu Y; Departments of Ophthalmology and Physiology, Feinberg School of Medicine, Northwestern University, Chicago, IL, 60611, USA.
Hande Özdinler P; Davee Department of Neurology and Clinical Neurological Sciences, Northwestern University, Feinberg School of Medicine, Chicago, IL, 60611, USA. .
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Źródło :
Gene therapy [Gene Ther] 2022 Apr; Vol. 29 (3-4), pp. 178-192. Date of Electronic Publication: 2021 Dec 02.
Typ publikacji :
Journal Article
MeSH Terms :
Amyotrophic Lateral Sclerosis*/genetics
Amyotrophic Lateral Sclerosis*/therapy
Animals ; DNA-Binding Proteins/genetics ; Disease Models, Animal ; Genetic Therapy ; Humans ; Mice ; Mice, Transgenic ; Motor Neurons/pathology ; Superoxide Dismutase/genetics ; Superoxide Dismutase-1/genetics ; Ubiquitin Thiolesterase/genetics
Czasopismo naukowe
Tytuł :
Getting the upper hand in ALS.
Autorzy :
Svendsen CN; Board of Governors Regenerative Medicine Institute Cedars-Sinai Medical Center, 8700 Beverly Blvd, AHSP 8th Floor, Los Angeles, CA, 90048, USA. .
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Źródło :
Gene therapy [Gene Ther] 2022 Apr; Vol. 29 (3-4), pp. 113-114. Date of Electronic Publication: 2022 Jan 20.
Typ publikacji :
Journal Article
MeSH Terms :
Amyotrophic Lateral Sclerosis*/genetics
Amyotrophic Lateral Sclerosis*/therapy
Humans
Czasopismo naukowe
Tytuł :
Membrane-bound MMP-14 protease-activatable adeno-associated viral vectors for gene delivery to pancreatic tumors.
Autorzy :
Butler SS; Department of Bioengineering, Rice University, Houston, TX, USA.
Date K; Departments of Translational Molecular Pathology and Pathology, Sheikh Ahmed Center for Pancreatic Cancer Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Okumura T; Departments of Translational Molecular Pathology and Pathology, Sheikh Ahmed Center for Pancreatic Cancer Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Lueck C; Department of Bioengineering, Rice University, Houston, TX, USA.
Ghosh B; Departments of Translational Molecular Pathology and Pathology, Sheikh Ahmed Center for Pancreatic Cancer Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Maitra A; Departments of Translational Molecular Pathology and Pathology, Sheikh Ahmed Center for Pancreatic Cancer Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Suh J; Department of Bioengineering, Rice University, Houston, TX, USA. .; Systems, Synthetic, and Physical Biology, Rice University, Houston, TX, USA. .
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Źródło :
Gene therapy [Gene Ther] 2022 Apr; Vol. 29 (3-4), pp. 138-146. Date of Electronic Publication: 2021 May 06.
Typ publikacji :
Journal Article
MeSH Terms :
Genetic Vectors*/genetics
Pancreatic Neoplasms*/genetics
Pancreatic Neoplasms*/therapy
Animals ; Dependovirus/metabolism ; Gene Transfer Techniques ; Matrix Metalloproteinase 14/genetics ; Matrix Metalloproteinases/genetics ; Mice ; Peptide Hydrolases/genetics
Czasopismo naukowe
Tytuł :
CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing.
Autorzy :
Yaméogo P; Centre de Recherche du CHU de Québec-Université Laval, Québec City, QC, Canada.; Département de Médecine Moléculaire, Université Laval, Québec City, QC, Canada.
Duchêne BL; Centre de Recherche du CHU de Québec-Université Laval, Québec City, QC, Canada.; Département de Médecine Moléculaire, Université Laval, Québec City, QC, Canada.
Majeau N; Centre de Recherche du CHU de Québec-Université Laval, Québec City, QC, Canada.; Département de Médecine Moléculaire, Université Laval, Québec City, QC, Canada.
Tremblay JP; Centre de Recherche du CHU de Québec-Université Laval, Québec City, QC, Canada. .; Département de Médecine Moléculaire, Université Laval, Québec City, QC, Canada. .
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Źródło :
Gene therapy [Gene Ther] 2022 Apr; Vol. 29 (3-4), pp. 171-177. Date of Electronic Publication: 2021 Oct 01.
Typ publikacji :
Journal Article
MeSH Terms :
CRISPR-Cas Systems*
Gene Editing*
CRISPR-Associated Protein 9/genetics ; Codon, Terminator/genetics ; HEK293 Cells ; Humans
Czasopismo naukowe
Tytuł :
AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1.
Autorzy :
Ozes B; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Myers M; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Moss K; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Mckinney J; Department of Pediatrics and Neurology, Nationwide Children's Hospital and The Ohio State University, Columbus, OH, USA.
Ridgley A; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Chen L; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Bai S; Department of Biomedical Informatics, The Ohio State University College of Medicine, Columbus, OH, USA.; Biostatistics Resource at Nationwide Children's Hospital, Columbus, OH, USA.
Abrams CK; Department of Neurology and Rehabilitation, University of Illinois at Chicago, Chicago, IL, USA.
Freidin MM; Department of Neurology and Rehabilitation, University of Illinois at Chicago, Chicago, IL, USA.
Mendell JR; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics and Neurology, Nationwide Children's Hospital and The Ohio State University, Columbus, OH, USA.
Sahenk Z; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA. .; Department of Pediatrics and Neurology, Nationwide Children's Hospital and The Ohio State University, Columbus, OH, USA. .; Department of Pathology and Laboratory Medicine, Nationwide Children's Hospital, Columbus, OH, USA. .
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Źródło :
Gene therapy [Gene Ther] 2022 Apr; Vol. 29 (3-4), pp. 127-137. Date of Electronic Publication: 2021 Feb 04.
Typ publikacji :
Journal Article
MeSH Terms :
Axons*
Charcot-Marie-Tooth Disease*/genetics
Charcot-Marie-Tooth Disease*/pathology
Charcot-Marie-Tooth Disease*/therapy
Animals ; Connexins/genetics ; Connexins/metabolism ; Genetic Therapy ; Humans ; Mice ; Mice, Knockout ; Mutation ; Nerve Regeneration ; Schwann Cells/metabolism
SCR Disease Name :
Charcot-Marie-Tooth disease, X-linked, 1
Czasopismo naukowe
Tytuł :
Non-viral delivery of CRISPR-Cas9 complexes for targeted gene editing via a polymer delivery system.
Autorzy :
O'Keeffe Ahern J; Charles Institute of Dermatology, University College Dublin, Dublin, Republic of Ireland.
Lara-Sáez I; Charles Institute of Dermatology, University College Dublin, Dublin, Republic of Ireland. .
Zhou D; Charles Institute of Dermatology, University College Dublin, Dublin, Republic of Ireland.
Murillas R; Epithelial Biomedicine Division, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Madrid, Spain.; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) ISCIII, Madrid, Spain.; Fundación Instituto de Investigaciones Sanitarias de la Fundación Jimenez Díaz, Madrid, Spain.
Bonafont J; Epithelial Biomedicine Division, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Madrid, Spain.; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) ISCIII, Madrid, Spain.; Fundación Instituto de Investigaciones Sanitarias de la Fundación Jimenez Díaz, Madrid, Spain.
Mencía Á; Epithelial Biomedicine Division, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Madrid, Spain.
García M; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) ISCIII, Madrid, Spain.; Fundación Instituto de Investigaciones Sanitarias de la Fundación Jimenez Díaz, Madrid, Spain.; Department of Bioengineering Universidad Carlos III de Madrid, Madrid, Spain.
Manzanares D; Charles Institute of Dermatology, University College Dublin, Dublin, Republic of Ireland.
Lynch J; Charles Institute of Dermatology, University College Dublin, Dublin, Republic of Ireland.
Foley R; Charles Institute of Dermatology, University College Dublin, Dublin, Republic of Ireland.
Xu Q; Charles Institute of Dermatology, University College Dublin, Dublin, Republic of Ireland.
Sigen A; Charles Institute of Dermatology, University College Dublin, Dublin, Republic of Ireland.
Larcher F; Epithelial Biomedicine Division, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Madrid, Spain.; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) ISCIII, Madrid, Spain.; Fundación Instituto de Investigaciones Sanitarias de la Fundación Jimenez Díaz, Madrid, Spain.; Department of Bioengineering Universidad Carlos III de Madrid, Madrid, Spain.
Wang W; Charles Institute of Dermatology, University College Dublin, Dublin, Republic of Ireland. .
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Źródło :
Gene therapy [Gene Ther] 2022 Apr; Vol. 29 (3-4), pp. 157-170. Date of Electronic Publication: 2021 Aug 06.
Typ publikacji :
Journal Article
MeSH Terms :
CRISPR-Cas Systems*
Epidermolysis Bullosa Dystrophica*/genetics
Epidermolysis Bullosa Dystrophica*/metabolism
Epidermolysis Bullosa Dystrophica*/therapy
Collagen Type VII/genetics ; Gene Editing ; HEK293 Cells ; Humans ; Polymers/metabolism
Czasopismo naukowe
Tytuł :
LncRNA SNHG16 promotes colorectal cancer cell proliferation, migration, and epithelial-mesenchymal transition through miR-124-3p/MCP-1.
Autorzy :
Chen ZY; Department of Gastroenterology, Hunan Provincial People's Hospital, The First Affiliated Hospital of Hunan Normal University, 410005, Changsha, P.R. China. .
Wang XY; Department of Gastroenterology, Hunan Provincial People's Hospital, The First Affiliated Hospital of Hunan Normal University, 410005, Changsha, P.R. China.
Yang YM; Department of Gastroenterology, Hunan Provincial People's Hospital, The First Affiliated Hospital of Hunan Normal University, 410005, Changsha, P.R. China.
Wu MH; Department of Gastroenterology, Hunan Provincial People's Hospital, The First Affiliated Hospital of Hunan Normal University, 410005, Changsha, P.R. China.
Yang L; Department of Gastroenterology, Hunan Provincial People's Hospital, The First Affiliated Hospital of Hunan Normal University, 410005, Changsha, P.R. China.
Jiang DT; Department of Gastroenterology, Hunan Provincial People's Hospital, The First Affiliated Hospital of Hunan Normal University, 410005, Changsha, P.R. China.
Cai H; Department of Gastroenterology, Hunan Provincial People's Hospital, The First Affiliated Hospital of Hunan Normal University, 410005, Changsha, P.R. China.
Peng Y; Department of Gastroenterology, Hunan Provincial People's Hospital, The First Affiliated Hospital of Hunan Normal University, 410005, Changsha, P.R. China. .
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Źródło :
Gene therapy [Gene Ther] 2022 Apr; Vol. 29 (3-4), pp. 193-205. Date of Electronic Publication: 2020 Aug 28.
Typ publikacji :
Journal Article
MeSH Terms :
Colorectal Neoplasms*/genetics
Colorectal Neoplasms*/pathology
MicroRNAs*/genetics
MicroRNAs*/metabolism
RNA, Long Noncoding*/genetics
RNA, Long Noncoding*/metabolism
Cell Movement/genetics ; Cell Proliferation/genetics ; Epithelial-Mesenchymal Transition/genetics ; Humans
Czasopismo naukowe

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