Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy - Wikidata - wikimedia - TriplyDB

Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy

Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy

http://www.wikidata.org/entity/Q36740284

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Current and emerging treatment strategies for Duchenne muscular dystrophy The emerging role of viral vectors as vehicles for DMD gene editing Intra-amniotic rAAV-mediated microdystrophin gene transfer improves canine X-linked muscular dystrophy and may induce immune tolerance Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy Partial restoration of cardiac function with ΔPDZ nNOS in aged mdx model of Duchenne cardiomyopathy.Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.The golden retriever model of Duchenne muscular dystrophy.The potential of adeno-associated viral vectors for gene delivery to muscle tissue.Progress and prospects of gene therapy clinical trials for the muscular dystrophies.Motor physical therapy affects muscle collagen type I and decreases gait speed in dystrophin-deficient dogs.AAV-8 is more efficient than AAV-9 in transducing neonatal dog heart.Duchenne muscular dystrophy gene therapy in the canine model.Porcine models of muscular dystrophy Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus.Gene Therapy for Duchenne muscular dystrophy.Perspective on Adeno-Associated Virus Capsid Modification for Duchenne Muscular Dystrophy Gene Therapy Exclusive skeletal muscle correction does not modulate dystrophic heart disease in the aged mdx model of Duchenne cardiomyopathy In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy.Therapeutic advances in muscular dystrophy Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model.Genomic removal of a therapeutic mini-dystrophin gene from adult mice elicits a Duchenne muscular dystrophy-like phenotype The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer.Dual AAV gene therapy for Duchenne muscular dystrophy with a 7-kb mini-dystrophin gene in the canine model.Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.Nanotherapy for Duchenne muscular dystrophy.Progress toward Gene Therapy for Duchenne Muscular Dystrophy.Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy.The Gene Therapy Resource Program: A Decade of Dedication to Translational Research by the National Heart, Lung, and Blood Institute.Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy.Inhibition of antigen presentation during AAV gene therapy using virus peptides.Micro-dystrophin gene therapy goes systemic in Duchenne muscular dystrophy patients.Treating pediatric neuromuscular disorders: The future is now.At the Crossroads of Clinical and Preclinical Research for Muscular Dystrophy-Are We Closer to Effective Treatment for Patients?Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy Gene and Cell Therapy for Muscular Dystrophies: Are We Getting There?

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Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy

http://www.wikidata.org/entity/Q36740284

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Molecular Therapy

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Microdystrophin ameliorates mu ...... of duchenne muscular dystrophy

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Chady H Hakim

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Hsiao T Yang

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Jin-Hong Shin

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Keqing Zhang

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Ronald L Terjung

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Xiufang Pan

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Yongping Yue

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P2860

A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy

Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs

An intronic LINE-1 element insertion in the dystrophin gene aborts dystrophin expression and results in Duchenne-like muscular dystrophy in the corgi breed

rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice.

The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins.

Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs.

Immunohistochemical analysis of dystrophin-associated proteins in Becker/Duchenne muscular dystrophy with huge in-frame deletions in the NH2-terminal and rod domains of dystrophin

Very mild muscular dystrophy associated with the deletion of 46% of dystrophin.

Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule.

Complementary DNA probes for the Duchenne muscular dystrophy locus demonstrate a previously undetectable deletion in a patient with dystrophic myopathy, glycerol kinase deficiency, and congenital adrenal hypoplasia

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