Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins.
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Follistatin gene delivery enhances muscle growth and strength in nonhuman primatesThe emerging role of viral vectors as vehicles for DMD gene editingHomologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transferTrendelenburg-Like Gait, Instability and Altered Step Patterns in a Mouse Model for Limb Girdle Muscular Dystrophy 2iTreatment of diabetes and long-term survival after insulin and glucokinase gene therapy.Sustained normalization of neurological disease after intracranial gene therapy in a feline model.Efficacy of Gene Therapy Is Dependent on Disease Progression in Dystrophic Mice with Mutations in the FKRP GeneAAV's anatomy: roadmap for optimizing vectors for translational successPersistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery.Immune responses to AAV in canine muscle monitored by cellular assays and noninvasive imaging.Influence of immune responses in gene/stem cell therapies for muscular dystrophies.Homozygous nonsense mutation in SGCA is a common cause of limb-girdle muscular dystrophy in Assiut, Egypt.Hoechst increases adeno-associated virus-mediated transgene expression in airway epithelia by inducing the cytomegalovirus promoterThe potential of adeno-associated viral vectors for gene delivery to muscle tissue.Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions.RNAi-based gene therapy for dominant Limb Girdle Muscular Dystrophies.Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.Gene therapy for muscular dystrophies: progress and challengesSustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.Gene therapy for inherited muscle diseases: where genetics meets rehabilitation medicine.A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.Progress and prospects of gene therapy clinical trials for the muscular dystrophies.Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors.Gene therapy for muscular dystrophy: moving the field forward.Effects of irradiating adult mdx mice before full-length dystrophin cDNA transfer on host anti-dystrophin immunityPhase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.B-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case StudyA dystrophic muscle broadens the contribution and activation of immune cells reacting to rAAV gene transfer.AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal ModelsSafety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophy.Recombinant adeno-associated viral vector production and purification.High-Pressure Transvenous Perfusion of the Upper Extremity in Human Muscular Dystrophy: A Safety Study with 0.9% Saline.RNA interference inhibits DUX4-induced muscle toxicity in vivo: implications for a targeted FSHD therapy.AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypesSuccessful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies.Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning.Gene therapy for muscular dystrophy: lessons learned and path forwardA GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB.Evading the immune response upon in vivo gene therapy with viral vectors.
P2860
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P2860
Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins.
description
2009 nî lūn-bûn
@nan
2009年の論文
@ja
2009年学术文章
@wuu
2009年学术文章
@zh
2009年学术文章
@zh-cn
2009年学术文章
@zh-hans
2009年学术文章
@zh-my
2009年学术文章
@zh-sg
2009年學術文章
@yue
2009年學術文章
@zh-hant
name
Limb-girdle muscular dystrophy ...... lycan and associated proteins.
@en
Limb-girdle muscular dystrophy ...... lycan and associated proteins.
@nl
type
label
Limb-girdle muscular dystrophy ...... lycan and associated proteins.
@en
Limb-girdle muscular dystrophy ...... lycan and associated proteins.
@nl
prefLabel
Limb-girdle muscular dystrophy ...... lycan and associated proteins.
@en
Limb-girdle muscular dystrophy ...... lycan and associated proteins.
@nl
P2093
P2860
P356
P1433
P1476
Limb-girdle muscular dystrophy ...... lycan and associated proteins.
@en
P2093
Barry J Byrne
Christopher M Walker
Christopher Shilling
Gloria Galloway
Janaiah Kota
Jerry R Mendell
Josepha M Craenen
K Reed Clark
Katherine J Campbell
Laurence Viollet
P2860
P304
P356
10.1002/ANA.21732
P577
2009-09-01T00:00:00Z