Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice.
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Dystrophin and the two related genetic diseases, Duchenne and Becker muscular dystrophiesPharmacology of manipulating lean body massMulti-parametric MRI at 14T for muscular dystrophy mice treated with AAV vector-mediated gene therapyIntra-amniotic rAAV-mediated microdystrophin gene transfer improves canine X-linked muscular dystrophy and may induce immune toleranceCurrent understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophyThe polyproline site in hinge 2 influences the functional capacity of truncated dystrophins.Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.Gene and cell-mediated therapies for muscular dystrophy.Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.Chronic losartan administration reduces mortality and preserves cardiac but not skeletal muscle function in dystrophic mice.Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs.Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice.Phosphorylation within the cysteine-rich region of dystrophin enhances its association with β-dystroglycan and identifies a potential novel therapeutic target for skeletal muscle wasting.Therapeutic approaches to muscular dystrophy.Duchenne muscular dystrophy gene therapy: Lost in translation?Transcription factor rational design improves directed differentiation of human mesenchymal stem cells into skeletal myocytes.AAV-microdystrophin therapy improves cardiac performance in aged female mdx mice.Duchenne muscular dystrophy gene therapy in the canine model.Porcine models of muscular dystrophyHuman galectin 3 binding protein interacts with recombinant adeno-associated virus type 6Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus.Evaluation of follistatin as a therapeutic in models of skeletal muscle atrophy associated with denervation and tenotomy.Combinatorial therapeutic activation with heparin and AICAR stimulates additive effects on utrophin A expression in dystrophic muscles.Truncated dystrophins reduce muscle stiffness in the extensor digitorum longus muscle of mdx mice.Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids.Progress in gene therapy of dystrophic heart disease.Cell-lineage regulated myogenesis for dystrophin replacement: a novel therapeutic approach for treatment of muscular dystrophyCurrent Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy.Gene therapy in large animal models of muscular dystrophy.Delivery of recombinant adeno-associated virus vectors to rat diaphragm muscle via direct intramuscular injectionCirculating Biomarkers for Duchenne Muscular Dystrophy.The function of dog models in developing gene therapy strategies for human health.New developments in the use of gene therapy to treat Duchenne muscular dystrophy.Drug Discovery of Therapies for Duchenne Muscular Dystrophy.Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.Systemic delivery of adeno-associated viral vectors.Gene therapy in myotubular myopathy: promising progress and future directions.Dystrophin and Spectrin, Two Highly Dissimilar Sisters of the Same Family.Nanotherapy for Duchenne muscular dystrophy.Delivery of AAV2/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of α1-syntrophin and α-dystrobrevin in skeletal muscles of mdx mice.
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Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice.
description
article científic
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article scientifique
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articolo scientifico
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artigo científico
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bilimsel makale
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scientific article published on 04 March 2008
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
Systemic microdystrophin gene ...... on in old dystrophic mdx mice.
@en
Systemic microdystrophin gene ...... on in old dystrophic mdx mice.
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type
label
Systemic microdystrophin gene ...... on in old dystrophic mdx mice.
@en
Systemic microdystrophin gene ...... on in old dystrophic mdx mice.
@nl
prefLabel
Systemic microdystrophin gene ...... on in old dystrophic mdx mice.
@en
Systemic microdystrophin gene ...... on in old dystrophic mdx mice.
@nl
P2860
P356
P1433
P1476
Systemic microdystrophin gene ...... ion in old dystrophic mdx mice
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P2093
James M Allen
Michael J Blankinship
P2860
P304
P356
10.1038/MT.2008.28
P577
2008-03-04T00:00:00Z