Informacja

Drogi użytkowniku, aplikacja do prawidłowego działania wymaga obsługi JavaScript. Proszę włącz obsługę JavaScript w Twojej przeglądarce.

Przeglądasz jako GOŚĆ

Wyszukujesz frazę ""MENDELL, JERRY R."" wg kryterium: Autor


Tytuł :
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.
Autorzy :
Day JW; Department of Neurology, Stanford University Medical Center, Palo Alto, CA, USA. Electronic address: .
Finkel RS; Department of Pediatrics, Nemours Children's Hospital, Orlando, FL, USA; Center for Experimental Neurotherapeutics, St Jude Children's Research Hospital, Memphis, TN, USA.
Chiriboga CA; Division of Pediatric Neurology, Columbia University Medical Center, New York, NY, USA.
Connolly AM; Department of Neurology, Nationwide Children's Hospital, Columbus, OH, USA; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics, Ohio State University, Columbus, OH, USA; Department of Neurology, Ohio State University, Columbus, OH, USA.
Crawford TO; Department of Neurology, Johns Hopkins Medicine, Baltimore, MD, USA; Department of Pediatrics, Johns Hopkins Medicine, Baltimore, MD, USA.
Darras BT; Department of Neurology, Boston Children's Hospital, Boston, MA, USA.
Iannaccone ST; Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Kuntz NL; Division of Neurology, Ann and Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA.
Peña LDM; Division of Human Genetics, Cincinnati Children's Hospital, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati OH, USA.
Shieh PB; Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
Smith EC; Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Kwon JM; Department of Neurology, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA.
Zaidman CM; Department of Neurology, Washington University School of Medicine, St Louis, MO, USA.
Schultz M; Novartis Gene Therapies, Bannockburn, IL, USA.
Feltner DE; Novartis Gene Therapies, Bannockburn, IL, USA.
Tauscher-Wisniewski S; Novartis Gene Therapies, Bannockburn, IL, USA.
Ouyang H; Novartis Gene Therapies, Bannockburn, IL, USA.
Chand DH; Department of Pediatrics, Washington University School of Medicine, St Louis, MO, USA; Novartis Gene Therapies, Bannockburn, IL, USA.
Sproule DM; Novartis Gene Therapies, Bannockburn, IL, USA.
Macek TA; Novartis Gene Therapies, Bannockburn, IL, USA.
Mendell JR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics, Ohio State University, Columbus, OH, USA; Department of Neurology, Ohio State University, Columbus, OH, USA.
Pokaż więcej
Źródło :
The Lancet. Neurology [Lancet Neurol] 2021 Apr; Vol. 20 (4), pp. 284-293. Date of Electronic Publication: 2021 Mar 17.
Typ publikacji :
Clinical Trial, Phase III; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't
MeSH Terms :
Biological Products/*therapeutic use
Genetic Therapy/*methods
Recombinant Fusion Proteins/*therapeutic use
Spinal Muscular Atrophies of Childhood/*drug therapy
Spinal Muscular Atrophies of Childhood/*genetics
Child, Preschool ; Female ; Humans ; Infant ; Male ; Survival of Motor Neuron 2 Protein/genetics ; Treatment Outcome
Czasopismo naukowe
Tytuł :
Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy.
Autorzy :
Willcocks RJ; Department of Physical Therapy, University of Florida, Gainesville.
Forbes SC; Department of Physical Therapy, University of Florida, Gainesville.
Walter GA; Department of Physical Therapy, University of Florida, Gainesville.
Sweeney L; Department of Physical Therapy, University of Florida, Gainesville.
Rodino-Klapac LR; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
Mendell JR; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Vandenborne K; Department of Physical Therapy, University of Florida, Gainesville.
Pokaż więcej
Źródło :
JAMA network open [JAMA Netw Open] 2021 Jan 04; Vol. 4 (1), pp. e2031851. Date of Electronic Publication: 2021 Jan 04.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, Non-P.H.S.
MeSH Terms :
Dystrophin/*genetics
Genetic Therapy/*methods
Magnetic Resonance Imaging/*methods
Muscular Dystrophy, Duchenne/*diagnostic imaging
Muscular Dystrophy, Duchenne/*therapy
Case-Control Studies ; Child ; Child, Preschool ; Humans ; Muscle, Skeletal/diagnostic imaging ; Treatment Outcome
Czasopismo naukowe
Tytuł :
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial.
Autorzy :
Mendell JR; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University, Columbus.; Department of Neurology, The Ohio State University, Columbus.
Sahenk Z; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University, Columbus.; Department of Neurology, The Ohio State University, Columbus.
Lehman K; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University, Columbus.
Nease C; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University, Columbus.
Lowes LP; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University, Columbus.; Department of Neurology, The Ohio State University, Columbus.
Miller NF; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Iammarino MA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Alfano LN; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Nicholl A; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Al-Zaidy S; Department of Pediatrics, The Ohio State University, Columbus.
Lewis S; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
Church K; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Shell R; Department of Pediatrics, The Ohio State University, Columbus.
Cripe LH; Department of Pediatrics, The Ohio State University, Columbus.
Potter RA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
Griffin DA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
Pozsgai E; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
Dugar A; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
Hogan M; Department of Radiology, Vascular and Interventional Radiology, Nationwide Children's Hospital, Columbus, Ohio.
Rodino-Klapac LR; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics, The Ohio State University, Columbus.; Sarepta Therapeutics Inc, Cambridge, Massachusetts.
Pokaż więcej
Źródło :
JAMA neurology [JAMA Neurol] 2020 Sep 01; Vol. 77 (9), pp. 1122-1131.
Typ publikacji :
Clinical Trial, Phase I; Clinical Trial, Phase II; Controlled Clinical Trial; Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms :
Dystrophin*/genetics
Outcome Assessment, Health Care*
Genetic Therapy/*methods
Muscular Dystrophy, Duchenne/*therapy
Child ; Child, Preschool ; Dependovirus ; Follow-Up Studies ; Gene Transfer Techniques ; Genetic Therapy/adverse effects ; Genetic Vectors ; Humans ; Male ; Muscle, Skeletal/metabolism ; Muscular Dystrophy, Duchenne/genetics ; Pilot Projects
Czasopismo naukowe
Tytuł :
Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes.
Autorzy :
Waldrop MA; Departments of Neurology and Pediatrics, The Ohio State University, Columbus, Ohio; .; Center for Gene Therapy and.
Karingada C; Departments of Neurology and.
Storey MA; Pharmacy, Nationwide Children's Hospital, Columbus, Ohio.
Powers B; Center for Gene Therapy and.
Iammarino MA; Center for Gene Therapy and.
Miller NF; Center for Gene Therapy and.
Alfano LN; Center for Gene Therapy and.
Noritz G; Department of Pediatrics, Nationwide Children's Hospital and The Ohio State University, Columbus, Ohio.
Rossman I; Departments of Pediatric Neurology and.
Ginsberg M; Departments of Pediatric Neurology and.
Mosher KA; Pediatric Physiatry, Akron Children's Hospital, Akron, Ohio.
Broomall E; Department of Pediatric Neurology, Cincinnati Children's Hospital, Cincinnati, Ohio; and.
Goldstein J; Division of Pediatric Neurology, Department of Pediatrics, Rainbow Babies and Children's Hospital, Cleveland, Ohio.
Bass N; Division of Pediatric Neurology, Department of Pediatrics, Rainbow Babies and Children's Hospital, Cleveland, Ohio.
Lowes LP; Center for Gene Therapy and.
Tsao CY; Departments of Neurology and Pediatrics, The Ohio State University, Columbus, Ohio.; Departments of Neurology and.
Mendell JR; Departments of Neurology and Pediatrics, The Ohio State University, Columbus, Ohio.; Center for Gene Therapy and.
Connolly AM; Departments of Neurology and Pediatrics, The Ohio State University, Columbus, Ohio.; Center for Gene Therapy and.
Pokaż więcej
Źródło :
Pediatrics [Pediatrics] 2020 Sep; Vol. 146 (3).
Typ publikacji :
Journal Article
MeSH Terms :
Genetic Therapy/*methods
Recombinant Fusion Proteins/*genetics
Spinal Muscular Atrophies of Childhood/*genetics
Spinal Muscular Atrophies of Childhood/*therapy
Survival of Motor Neuron 1 Protein/*genetics
Adenoviruses, Human ; Age Factors ; Alanine Transaminase/metabolism ; Aspartate Aminotransferases/metabolism ; Biological Products ; Genetic Therapy/adverse effects ; Genetic Vectors/administration & dosage ; Glucocorticoids/administration & dosage ; Humans ; Infant ; Ohio ; Outcome Assessment, Health Care ; Prednisolone/administration & dosage ; gamma-Glutamyltransferase/metabolism
Czasopismo naukowe
Tytuł :
Translational Medicine: Evolution, Fulfillment, and Belief in Gene Therapy.
Autorzy :
Mendell JR; Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio.; Department of Pediatrics and Neurology, The Ohio State University, Columbus, Ohio.
Pokaż więcej
Źródło :
Human gene therapy [Hum Gene Ther] 2020 Jan; Vol. 31 (1-2), pp. 14-19.
Typ publikacji :
Biography; Historical Article; Letter
MeSH Terms :
Genetic Therapy*/history
Genetic Therapy*/methods
Genetic Therapy*/trends
Translational Medical Research*/history
Translational Medical Research*/methods
Translational Medical Research*/trends
History, 20th Century ; Humans
Opinia redakcyjna
Tytuł :
From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1.
Autorzy :
Al-Zaidy SA; Department of Pediatrics, Ohio State University, Columbus, Ohio; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
Mendell JR; Department of Pediatrics, Ohio State University, Columbus, Ohio; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio; Department of Neurology, Ohio State University, Columbus, Ohio. Electronic address: jerry.mendell@nationwidechildrens.org.
Pokaż więcej
Źródło :
Pediatric neurology [Pediatr Neurol] 2019 Nov; Vol. 100, pp. 3-11. Date of Electronic Publication: 2019 Jun 13.
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't; Review
MeSH Terms :
Clinical Trials as Topic*
Genetic Therapy*
Survival of Motor Neuron 1 Protein*
Spinal Muscular Atrophies of Childhood/*therapy
Humans ; Spinal Muscular Atrophies of Childhood/genetics
Czasopismo naukowe
Tytuł :
Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion.
Autorzy :
Mendell JR; 1Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; 2Department of Pediatrics, The Ohio State University, Columbus, Ohio.; 3Department of Neurology, The Ohio State University, Columbus, Ohio.
Chicoine LG; 1Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; 2Department of Pediatrics, The Ohio State University, Columbus, Ohio.
Al-Zaidy SA; 2Department of Pediatrics, The Ohio State University, Columbus, Ohio.
Sahenk Z; 1Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; 2Department of Pediatrics, The Ohio State University, Columbus, Ohio.; 3Department of Neurology, The Ohio State University, Columbus, Ohio.
Lehman K; 1Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Lowes L; 1Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; 2Department of Pediatrics, The Ohio State University, Columbus, Ohio.; 3Department of Neurology, The Ohio State University, Columbus, Ohio.
Miller N; 1Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Alfano L; 1Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; 3Department of Neurology, The Ohio State University, Columbus, Ohio.
Galliers B; 1Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Lewis S; 1Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Murrey D; 1Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Peterson E; 1Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Griffin DA; 1Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Church K; 1Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Cheatham S; 4Department of Interventional Cardiology, The Ohio State University, Columbus, Ohio.
Cheatham J; 4Department of Interventional Cardiology, The Ohio State University, Columbus, Ohio.
Hogan MJ; 5Department of Radiology, Vascular and Interventional Radiology, Nationwide Children's Hospital, Columbus, Ohio.
Rodino-Klapac LR; 1Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio.; 2Department of Pediatrics, The Ohio State University, Columbus, Ohio.
Pokaż więcej
Źródło :
Human gene therapy [Hum Gene Ther] 2019 Jul; Vol. 30 (7), pp. 794-801. Date of Electronic Publication: 2019 Apr 19.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
MeSH Terms :
Gene Transfer Techniques*
Genetic Therapy*
Transgenes*
Genetic Vectors/*genetics
Muscular Dystrophies, Limb-Girdle/*genetics
Muscular Dystrophies, Limb-Girdle/*therapy
Sarcoglycanopathies/*genetics
Animals ; Biomarkers ; Child ; Disease Models, Animal ; Female ; Gene Expression ; Genetic Vectors/administration & dosage ; Humans ; Injections, Intramuscular ; Male ; Middle Aged ; Muscular Dystrophies, Limb-Girdle/physiopathology ; Transduction, Genetic ; Treatment Outcome
Czasopismo naukowe
Tytuł :
Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy.
Autorzy :
Al-Zaidy S; Department of Pediatrics, Ohio State University, Columbus, Ohio.; Center for Gene Therapy Nationwide Children's Hospital, Columbus, Ohio.
Pickard AS; Department of Pharmacy Systems, Outcomes, and Policy, University of Illinois, Chicago, Illinois.
Kotha K; Center for Gene Therapy Nationwide Children's Hospital, Columbus, Ohio.
Alfano LN; Department of Pediatrics, Ohio State University, Columbus, Ohio.; Center for Gene Therapy Nationwide Children's Hospital, Columbus, Ohio.
Lowes L; Department of Pediatrics, Ohio State University, Columbus, Ohio.; Center for Gene Therapy Nationwide Children's Hospital, Columbus, Ohio.
Paul G; Center for Gene Therapy Nationwide Children's Hospital, Columbus, Ohio.
Church K; Department of Pediatrics, Ohio State University, Columbus, Ohio.; Center for Gene Therapy Nationwide Children's Hospital, Columbus, Ohio.
Lehman K; Center for Gene Therapy Nationwide Children's Hospital, Columbus, Ohio.
Sproule DM; AveXis, Inc., Bannockburn, Illinois.
Dabbous O; AveXis, Inc., Bannockburn, Illinois.
Maru B; AveXis, Inc., Bannockburn, Illinois.
Berry K; Department of Pediatrics, Ohio State University, Columbus, Ohio.; Center for Gene Therapy Nationwide Children's Hospital, Columbus, Ohio.
Arnold WD; Department of Neurology, Ohio State University, Columbus, Ohio.
Kissel JT; Department of Pediatrics, Ohio State University, Columbus, Ohio.
Mendell JR; Department of Pediatrics, Ohio State University, Columbus, Ohio.; Center for Gene Therapy Nationwide Children's Hospital, Columbus, Ohio.; Department of Neurology, Ohio State University, Columbus, Ohio.
Shell R; Department of Pediatrics, Ohio State University, Columbus, Ohio.
Pokaż więcej
Źródło :
Pediatric pulmonology [Pediatr Pulmonol] 2019 Feb; Vol. 54 (2), pp. 179-185. Date of Electronic Publication: 2018 Dec 12.
Typ publikacji :
Clinical Trial; Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms :
Genetic Therapy*
Spinal Muscular Atrophies of Childhood/*therapy
Child, Preschool ; Humans ; Infant ; Infant, Newborn ; Mutation ; Spinal Muscular Atrophies of Childhood/genetics ; Survival of Motor Neuron 1 Protein/genetics ; Treatment Outcome
Czasopismo naukowe
Tytuł :
AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort.
Autorzy :
Al-Zaidy SA; Center for Gene Therapy Research Institute, Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University Wexner Medical Center, Columbus, OH, USA.
Kolb SJ; Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, OH, USA.; Department of Biological Chemistry and Pharmacology, The Ohio State University Wexner Medical Center, Columbus, OH, USA.
Lowes L; Center for Gene Therapy Research Institute, Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University Wexner Medical Center, Columbus, OH, USA.
Alfano LN; Center for Gene Therapy Research Institute, Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University Wexner Medical Center, Columbus, OH, USA.
Shell R; Department of Pediatrics, The Ohio State University Wexner Medical Center, Columbus, OH, USA.
Church KR; Center for Gene Therapy Research Institute, Nationwide Children's Hospital, Columbus, OH, USA.
Nagendran S; AveXis, Inc., Bannockburn, IL, USA.
Sproule DM; AveXis, Inc., Bannockburn, IL, USA.
Feltner DE; AveXis, Inc., Bannockburn, IL, USA.
Wells C; AveXis, Inc., Bannockburn, IL, USA.
Ogrinc F; AveXis, Inc., Bannockburn, IL, USA.
Menier M; AveXis, Inc., Bannockburn, IL, USA.
L'Italien J; AveXis, Inc., Bannockburn, IL, USA.
Arnold WD; Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, OH, USA.
Kissel JT; Department of Pediatrics, The Ohio State University Wexner Medical Center, Columbus, OH, USA.
Kaspar BK; AveXis, Inc., Bannockburn, IL, USA.
Mendell JR; Center for Gene Therapy Research Institute, Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University Wexner Medical Center, Columbus, OH, USA.; Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, OH, USA.
Pokaż więcej
Źródło :
Journal of neuromuscular diseases [J Neuromuscul Dis] 2019; Vol. 6 (3), pp. 307-317.
Typ publikacji :
Journal Article
MeSH Terms :
Genetic Therapy*
Spinal Muscular Atrophies of Childhood/*therapy
Female ; Humans ; Infant ; Kaplan-Meier Estimate ; Male ; Prospective Studies ; Spinal Muscular Atrophies of Childhood/genetics ; Treatment Outcome
Czasopismo naukowe
Tytuł :
Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy.
Autorzy :
Potter RA; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.
Griffin DA; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.
Sondergaard PC; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.
Johnson RW; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.
Pozsgai ER; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.; 3 Integrated Biomedical Science Graduate Program, College of Medicine, The Ohio State University , Columbus, Ohio; The Ohio State University, Columbus, Ohio.
Heller KN; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.
Peterson EL; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.
Lehtimäki KK; 5 Charles River Discovery Services , Kuopio, Finland .
Windish HP; 4 The Jain Foundation , Seattle, Washington.
Mittal PJ; 4 The Jain Foundation , Seattle, Washington.
Albrecht DE; 4 The Jain Foundation , Seattle, Washington.
Mendell JR; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.; 2 Department of Pediatrics and Neurology, The Ohio State University , Columbus, Ohio; The Ohio State University, Columbus, Ohio.
Rodino-Klapac LR; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.; 2 Department of Pediatrics and Neurology, The Ohio State University , Columbus, Ohio; The Ohio State University, Columbus, Ohio.; 3 Integrated Biomedical Science Graduate Program, College of Medicine, The Ohio State University , Columbus, Ohio; The Ohio State University, Columbus, Ohio.
Pokaż więcej
Źródło :
Human gene therapy [Hum Gene Ther] 2018 Jul; Vol. 29 (7), pp. 749-762. Date of Electronic Publication: 2017 Jul 13.
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms :
Genetic Therapy*
DNA, Complementary/*administration & dosage
Dysferlin/*genetics
Muscular Dystrophies, Limb-Girdle/*therapy
Animals ; DNA, Complementary/genetics ; Dependovirus/genetics ; Disease Models, Animal ; Dysferlin/administration & dosage ; Gene Expression Regulation ; Genetic Vectors/therapeutic use ; Humans ; Male ; Mice ; Muscle, Skeletal ; Muscular Dystrophies, Limb-Girdle/genetics ; Muscular Dystrophies, Limb-Girdle/pathology ; Mutation
SCR Disease Name :
Dysferlinopathy
Czasopismo naukowe
Tytuł :
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
Autorzy :
Mendell JR; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Al-Zaidy S; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Shell R; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Arnold WD; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Rodino-Klapac LR; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Prior TW; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Lowes L; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Alfano L; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Berry K; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Church K; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Kissel JT; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Nagendran S; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
L'Italien J; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Sproule DM; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Wells C; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Cardenas JA; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Heitzer MD; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Kaspar A; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Corcoran S; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Braun L; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Likhite S; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Miranda C; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Meyer K; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Foust KD; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Burghes AHM; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Kaspar BK; From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).
Pokaż więcej
Źródło :
The New England journal of medicine [N Engl J Med] 2017 Nov 02; Vol. 377 (18), pp. 1713-1722.
Typ publikacji :
Clinical Trial, Phase I; Clinical Trial, Phase II; Controlled Clinical Trial; Journal Article
MeSH Terms :
Genetic Therapy*/adverse effects
Spinal Muscular Atrophies of Childhood/*therapy
Survival of Motor Neuron 1 Protein/*genetics
Cohort Studies ; Dependovirus ; Disease-Free Survival ; Female ; Genetic Vectors ; Historically Controlled Study ; Humans ; Infant ; Infant, Newborn ; Infusions, Intravenous ; Liver Diseases/etiology ; Male ; Motor Skills ; Nutritional Support ; Respiration, Artificial ; Spinal Muscular Atrophies of Childhood/genetics ; Spinal Muscular Atrophies of Childhood/physiopathology
Czasopismo naukowe
Tytuł :
Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice.
Autorzy :
Voit A; Department of Cell Biology and Molecular Medicine, New Jersey Medical School, Rutgers, The State University of New Jersey, Newark, NJ, 07103, USA.
Patel V; Department of Pharmacology, Physiology and Neuroscience, New Jersey Medical School, Rutgers, The State University of New Jersey, Newark, NJ, 07103, USA.
Pachon R; Department of Cell Biology and Molecular Medicine, New Jersey Medical School, Rutgers, The State University of New Jersey, Newark, NJ, 07103, USA.
Shah V; Department of Cell Biology and Molecular Medicine, New Jersey Medical School, Rutgers, The State University of New Jersey, Newark, NJ, 07103, USA.
Bakhutma M; Department of Cell Biology and Molecular Medicine, New Jersey Medical School, Rutgers, The State University of New Jersey, Newark, NJ, 07103, USA.
Kohlbrenner E; Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, 10029, USA.
McArdle JJ; Department of Pharmacology, Physiology and Neuroscience, New Jersey Medical School, Rutgers, The State University of New Jersey, Newark, NJ, 07103, USA.
Dell'Italia LJ; Department of Medicine, University of Alabama at Birmingham, and Birmingham VA Medical Center, Birmingham, AL, 35294, USA.
Mendell JR; Department of Pediatrics and Department of Neurology, Ohio State University Research Institute at Nationwide Children's Hospital, Columbus, OH, 43205, USA.
Xie LH; Department of Cell Biology and Molecular Medicine, New Jersey Medical School, Rutgers, The State University of New Jersey, Newark, NJ, 07103, USA.
Hajjar RJ; Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, 10029, USA.
Duan D; Department of Molecular Microbiology and Immunology, Neurology, Bioengineering, Biomedical Sciences, The University of Missouri, Columbia, MO, 65212, USA.
Fraidenraich D; Department of Cell Biology and Molecular Medicine, New Jersey Medical School, Rutgers, The State University of New Jersey, Newark, NJ, 07103, USA.
Babu GJ; Department of Cell Biology and Molecular Medicine, New Jersey Medical School, Rutgers, The State University of New Jersey, Newark, NJ, 07103, USA. .
Pokaż więcej
Źródło :
Nature communications [Nat Commun] 2017 Oct 20; Vol. 8 (1), pp. 1068. Date of Electronic Publication: 2017 Oct 20.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
MeSH Terms :
Gene Silencing*
Genetic Therapy*
Cardiomyopathies/*physiopathology
Gene Expression Regulation/*genetics
Muscle Proteins/*genetics
Muscular Dystrophy, Duchenne/*physiopathology
Muscular Dystrophy, Duchenne/*therapy
Proteolipids/*genetics
Animals ; Cardiomyopathies/genetics ; Disease Models, Animal ; Dystrophin/genetics ; Dystrophin/metabolism ; Mice ; Mice, Inbred mdx ; Mice, Knockout ; Muscle Proteins/metabolism ; Muscular Dystrophy, Duchenne/genetics ; Proteolipids/metabolism ; RNA Interference ; Sarcoplasmic Reticulum Calcium-Transporting ATPases/genetics ; Sarcoplasmic Reticulum Calcium-Transporting ATPases/metabolism ; Utrophin/genetics ; Utrophin/metabolism
Czasopismo naukowe
Tytuł :
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes.
Autorzy :
Mendell JR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA. Electronic address: jerry.mendell@nationwidechildrens.org.
Sahenk Z; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA.
Al-Zaidy S; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA.
Rodino-Klapac LR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA.
Lowes LP; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA; Clinical Therapies, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Alfano LN; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA; Clinical Therapies, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Berry K; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA; Clinical Therapies, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Miller N; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA; Clinical Therapies, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Yalvac M; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Dvorchik I; Biostatics Research Core, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Moore-Clingenpeel M; Biostatics Research Core, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Flanigan KM; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA.
Church K; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Shontz K; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Curry C; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Lewis S; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
McColly M; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Hogan MJ; Vascular and Interventional Radiology, Department of Radiology, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Kaspar BK; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA.
Pokaż więcej
Źródło :
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2017 Apr 05; Vol. 25 (4), pp. 870-879. Date of Electronic Publication: 2017 Mar 06.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
MeSH Terms :
Genetic Therapy*/methods
Follistatin/*genetics
Myositis, Inclusion Body/*genetics
Myositis, Inclusion Body/*therapy
AMP-Activated Protein Kinases/metabolism ; Aged ; Animals ; Biomarkers ; Biopsy ; Dependovirus/genetics ; Dependovirus/immunology ; Follow-Up Studies ; Gene Dosage ; Genetic Vectors/administration & dosage ; Genetic Vectors/adverse effects ; Genetic Vectors/genetics ; Humans ; Male ; Mice ; Middle Aged ; Muscle, Skeletal/metabolism ; Muscle, Skeletal/pathology ; Muscle, Skeletal/physiopathology ; Myositis, Inclusion Body/diagnosis ; Recovery of Function ; TOR Serine-Threonine Kinases/metabolism ; Treatment Outcome ; Walk Test
Czasopismo naukowe
Tytuł :
Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.
Autorzy :
Findlay AR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH.
Wein N
Kaminoh Y
Taylor LE
Dunn DM
Mendell JR
King WM
Pestronk A
Florence JM
Mathews KD
Finkel RS
Swoboda KJ
Howard MT
Day JW
McDonald C
Nicolas A
Le Rumeur E
Weiss RB
Flanigan KM
Pokaż więcej
Corporate Authors :
United Dystrophinopathy Project
Źródło :
Annals of neurology [Ann Neurol] 2015 Apr; Vol. 77 (4), pp. 668-74. Date of Electronic Publication: 2015 Mar 02.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
MeSH Terms :
Databases, Genetic*
Phenotype*
Exons/*genetics
Genetic Therapy/*methods
Muscular Dystrophy, Duchenne/*genetics
Muscular Dystrophy, Duchenne/*therapy
Adolescent ; Adult ; Aged ; Child ; Child, Preschool ; Cohort Studies ; Humans ; Male ; Middle Aged ; Muscular Dystrophy, Duchenne/diagnosis ; Predictive Value of Tests ; Treatment Outcome ; Young Adult
Czasopismo naukowe
Tytuł :
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.
Autorzy :
Meyer K; The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Ferraiuolo L; The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Schmelzer L; The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Braun L; The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
McGovern V; Department of Molecular & Cellular Biochemistry, The Ohio State University Medical Center, Columbus, Ohio, USA.
Likhite S; 1] The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA [2] Molecular, Cellular & Developmental Biology Graduate Program, The Ohio State University, Columbus, Ohio, USA.
Michels O; The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Govoni A; The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Fitzgerald J; Department of Neuroscience, The Ohio State University, Columbus, Ohio, USA.
Morales P; Mannheimer Foundation, Inc., Homestead, Florida, USA.
Foust KD; Department of Neuroscience, The Ohio State University, Columbus, Ohio, USA.
Mendell JR; 1] The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA [2] Molecular, Cellular & Developmental Biology Graduate Program, The Ohio State University, Columbus, Ohio, USA [3] Department of Neuroscience, The Ohio State University, Columbus, Ohio, USA.
Burghes AH; Department of Molecular & Cellular Biochemistry, The Ohio State University Medical Center, Columbus, Ohio, USA.
Kaspar BK; 1] The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA [2] Molecular, Cellular & Developmental Biology Graduate Program, The Ohio State University, Columbus, Ohio, USA [3] Department of Neuroscience, The Ohio State University, Columbus, Ohio, USA.
Pokaż więcej
Źródło :
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2015 Mar; Vol. 23 (3), pp. 477-87. Date of Electronic Publication: 2014 Oct 31.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
MeSH Terms :
Dependovirus/*genetics
Genetic Therapy/*methods
Genetic Vectors/*administration & dosage
Muscular Atrophy, Spinal/*therapy
Spinal Cord/*metabolism
Survival of Motor Neuron 1 Protein/*genetics
Animals ; Animals, Newborn ; Brain Stem/metabolism ; Cerebral Cortex/metabolism ; DNA, Complementary/administration & dosage ; DNA, Complementary/genetics ; DNA, Complementary/metabolism ; Disease Models, Animal ; Dose-Response Relationship, Drug ; Gene Expression ; Genetic Vectors/pharmacokinetics ; Injections, Epidural ; Macaca fascicularis ; Mice ; Mice, Knockout ; Motor Neurons/metabolism ; Motor Neurons/pathology ; Muscular Atrophy, Spinal/genetics ; Muscular Atrophy, Spinal/metabolism ; Muscular Atrophy, Spinal/pathology ; Spinal Cord/pathology ; Survival of Motor Neuron 1 Protein/metabolism ; Transduction, Genetic ; Transgenes
Czasopismo naukowe
Tytuł :
Perspectives on best practices for gene therapy programs.
Autorzy :
Cheever TR; 1 National Institute of Neurological Disorders and Stroke, National Institutes of Health , Bethesda, MD 20892.
Berkley D
Braun S
Brown RH
Byrne BJ
Chamberlain JS
Cwik V
Duan D
Federoff HJ
High KA
Kaspar BK
Klinger KW
Larkindale J
Lincecum J
Mavilio F
McDonald CL
McLaughlin J
Weiss McLeod B
Mendell JR
Nuckolls G
Stedman HH
Tagle DA
Vandenberghe LH
Wang H
Wernett PJ
Wilson JM
Porter JD
Gubitz AK
Pokaż więcej
Źródło :
Human gene therapy [Hum Gene Ther] 2015 Mar; Vol. 26 (3), pp. 127-33. Date of Electronic Publication: 2015 Mar 03.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
MeSH Terms :
Genetic Therapy/*methods
Genetic Therapy/*standards
Neuromuscular Diseases/*genetics
Neuromuscular Diseases/*therapy
Clinical Trials as Topic ; Genetic Therapy/legislation & jurisprudence ; Government Regulation ; Humans ; Intellectual Property
Czasopismo naukowe
Tytuł :
A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.
Autorzy :
Mendell JR; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA [3] Department of Neurology, The Ohio State University, Columbus, Ohio, USA.
Sahenk Z; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA [3] Department of Neurology, The Ohio State University, Columbus, Ohio, USA.
Malik V; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
Gomez AM; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
Flanigan KM; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA [3] Department of Neurology, The Ohio State University, Columbus, Ohio, USA.
Lowes LP; 1] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA [2] Department of Physical Medicine and Rehabilitation, The Ohio State University, Columbus, Ohio, USA.
Alfano LN; 1] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA [2] Department of Physical Medicine and Rehabilitation, The Ohio State University, Columbus, Ohio, USA.
Berry K; 1] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA [2] Department of Physical Medicine and Rehabilitation, The Ohio State University, Columbus, Ohio, USA.
Meadows E; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
Lewis S; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
Braun L; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
Shontz K; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
Rouhana M; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
Clark KR; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
Rosales XQ; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
Al-Zaidy S; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
Govoni A; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
Rodino-Klapac LR; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
Hogan MJ; Department of Radiology, Vascular and Interventional Radiology, Nationwide Children's Hospital, Columbus, Ohio, USA.
Kaspar BK; 1] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
Pokaż więcej
Źródło :
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2015 Jan; Vol. 23 (1), pp. 192-201. Date of Electronic Publication: 2014 Oct 17.
Typ publikacji :
Clinical Trial, Phase I; Clinical Trial, Phase II; Journal Article; Research Support, N.I.H., Extramural
MeSH Terms :
Dystrophin/*deficiency
Follistatin-Related Proteins/*genetics
Genetic Therapy/*methods
Muscular Dystrophy, Duchenne/*therapy
Myostatin/*genetics
Adult ; Dependovirus/genetics ; Dystrophin/genetics ; Follistatin-Related Proteins/metabolism ; Gene Expression ; Genetic Vectors ; Humans ; Injections, Intramuscular ; Male ; Muscle, Skeletal/metabolism ; Muscle, Skeletal/pathology ; Muscular Dystrophy, Duchenne/genetics ; Muscular Dystrophy, Duchenne/metabolism ; Muscular Dystrophy, Duchenne/pathology ; Myostatin/antagonists & inhibitors ; Myostatin/metabolism
Czasopismo naukowe
Tytuł :
Gene therapy for muscular dystrophy: moving the field forward.
Autorzy :
Al-Zaidy S; Department of Pediatrics, Center for Gene Therapy, The Research Institute of Nationwide Children's Hospital, Columbus, Ohio.
Rodino-Klapac L; Department of Pediatrics, Center for Gene Therapy, The Research Institute of Nationwide Children's Hospital, Columbus, Ohio.
Mendell JR; Department of Pediatrics, Center for Gene Therapy, The Research Institute of Nationwide Children's Hospital, Columbus, Ohio. Electronic address: Jerry.Mendell@nationwidechildrens.org.
Pokaż więcej
Źródło :
Pediatric neurology [Pediatr Neurol] 2014 Nov; Vol. 51 (5), pp. 607-18. Date of Electronic Publication: 2014 Aug 07.
Typ publikacji :
Journal Article; Review
MeSH Terms :
Genetic Therapy/*methods
Genetic Therapy/*trends
Muscular Dystrophies/*genetics
Muscular Dystrophies/*therapy
Animals ; Humans
Czasopismo naukowe
Tytuł :
Charting a clear path: the ASGCT Standardized Pathways Conference.
Autorzy :
Kiem HP; Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Department of Medicine, University of Washington, Seattle, Washington, USA. Electronic address: .
Baum C; Department of Experimental Hematology, Hannover Medical School, Hannover, Germany.
Bushman FD; Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA.
Byrne BJ; Department of Pediatrics, Divisions of Cellular and Molecular Therapy and Pediatric Cardiology, College of Medicine, University of Florida, Gainesville, Florida, USA; Powell Gene Therapy Center, University of Florida, Gainesville, Florida, USA.
Carter BJ; BioMarin Pharmaceutical, San Rafael, California, USA.
Cavagnaro J; Access BIO, Boyce, Virginia, USA.
Malech HL; Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.
Mendell JR; Paul D. Wellstone Center, Nationwide Children's Hospital and The Ohio State University, Columbus, Ohio, USA.
Naldini LM; Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Scientific Institute, San Raffaele Telethon Institute for Gene Therapy (TIGET), Milan, Italy.
Sorrentino BP; Division of Experimental Hematology, Department of Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Williams DA; Hematology/Oncology Division, Children's Hospital Boston and Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts, USA.
Flotte TR; Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA.
Pokaż więcej
Źródło :
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2014 Jul; Vol. 22 (7), pp. 1235-1238.
Typ publikacji :
Journal Article
MeSH Terms :
Genetic Therapy*
Congresses as Topic ; Genetic Vectors ; Humans
Czasopismo naukowe
Tytuł :
Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin α2 surrogates.
Autorzy :
Chicoine LG; 1] Department of Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [2] Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Rodino-Klapac LR; 1] Department of Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [2] Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Shao G; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Xu R; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Bremer WG; Vaccines and Immunity, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Camboni M; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Golden B; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Montgomery CL; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Shontz K; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Heller KN; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Griffin DA; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Lewis S; Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Coley BD; 1] Department of Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [2] Current address: Department of Radiology, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA.
Walker CM; 1] Department of Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [2] Vaccines and Immunity, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Clark KR; 1] Department of Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [2] Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Sahenk Z; 1] Department of Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Neurology, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [3] Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Mendell JR; 1] Department of Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [2] Department of Neurology, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [3] Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Martin PT; 1] Department of Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio, USA [2] Centers for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
Pokaż więcej
Źródło :
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2014 Apr; Vol. 22 (4), pp. 713-24. Date of Electronic Publication: 2013 Oct 22.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
MeSH Terms :
Gene Transfer Techniques*
Genetic Therapy*
Dystrophin/*biosynthesis
Laminin/*biosynthesis
Muscular Dystrophies/*genetics
Animals ; Dependovirus/genetics ; Disease Models, Animal ; Dystroglycans/genetics ; Dystroglycans/metabolism ; Dystrophin/genetics ; Gene Expression Regulation ; Glycosyltransferases/genetics ; Laminin/genetics ; Macaca mulatta/genetics ; Muscle, Skeletal/metabolism ; Muscle, Skeletal/pathology ; Muscular Dystrophies/pathology ; Muscular Dystrophies/therapy
Czasopismo naukowe

Ta witryna wykorzystuje pliki cookies do przechowywania informacji na Twoim komputerze. Pliki cookies stosujemy w celu świadczenia usług na najwyższym poziomie, w tym w sposób dostosowany do indywidualnych potrzeb. Korzystanie z witryny bez zmiany ustawień dotyczących cookies oznacza, że będą one zamieszczane w Twoim komputerze. W każdym momencie możesz dokonać zmiany ustawień dotyczących cookies