Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. - Wikidata - awgldk - TriplyDB

Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy.

Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy.

http://www.wikidata.org/entity/Q45883916

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Cell membrane expression of cardiac sodium channel Na(v)1.5 is modulated by alpha-actinin-2 interaction Follistatin gene delivery enhances muscle growth and strength in nonhuman primates Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials Dystrophin and the two related genetic diseases, Duchenne and Becker muscular dystrophies Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer Inhibitory control over Ca(2+) sparks via mechanosensitive channels is disrupted in dystrophin deficient muscle but restored by mini-dystrophin expression The Crystal Structures of Dystrophin and Utrophin Spectrin Repeats: Implications for Domain Boundaries Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy Muscular dystrophy begins early in embryonic development deriving from stem cell loss and disrupted skeletal muscle formation Report on the 3rd Ottawa International Conference on Neuromuscular Biology, Disease and Therapy - September 24-26, 2015, Ottawa, Canada.The Dystrophin Complex: Structure, Function, and Implications for Therapy Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice Skeletal muscles of ambulant children with Duchenne muscular dystrophy: validation of multicenter study of evaluation with MR imaging and MR spectroscopy.rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice.Interaction of dystrophin rod domain with membrane phospholipids. Evidence of a close proximity between tryptophan residues and lipids.Systemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cells Enhanced effect of microdystrophin gene transfection by HSV-VP22 mediated intercellular protein transport L-type Ca2+ channel function is linked to dystrophin expression in mammalian muscle Skeletal muscle-specific ablation of gamma(cyto)-actin does not exacerbate the mdx phenotype.Genetic correction of splice site mutation in purified and enriched myoblasts isolated from mdx5cv mice.The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins.MicroRNA-29 overexpression by adeno-associated virus suppresses fibrosis and restores muscle function in combination with micro-dystrophin Truncated dystrophins can influence neuromuscular synapse structure.Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.Analysis of dystrophin deletion mutations predicts age of cardiomyopathy onset in becker muscular dystrophy.Systemic Trans-splicing adeno-associated viral delivery efficiently transduces the heart of adult mdx mouse, a model for duchenne muscular dystrophy Dystrophin delivery to muscles of mdx mice using lentiviral vectors leads to myogenic progenitor targeting and stable gene expression.Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery.Full-length dystrophin reconstitution with adeno-associated viral vectors MLPA identification of dystrophin mutations and in silico evaluation of the predicted protein in dystrophinopathy cases from India.Emerging genetic therapies to treat Duchenne muscular dystrophy Gene and cell-mediated therapies for muscular dystrophy.Transfer of the full-length dystrophin-coding sequence into muscle cells by a dual high-capacity hybrid viral vector with site-specific integration ability DUX4, a candidate gene for facioscapulohumeral muscular dystrophy, causes p53-dependent myopathy in vivo Adeno-associated virus serotype-9 microdystrophin gene therapy ameliorates electrocardiographic abnormalities in mdx mice.Neuronal nitric oxide synthase-rescue of dystrophin/utrophin double knockout mice does not require nNOS localization to the cell membrane Restoration of dystrophin expression using the Sleeping Beauty transposon.Magnetic resonance imaging and spectroscopy assessment of lower extremity skeletal muscles in boys with Duchenne muscular dystrophy: a multicenter cross sectional study A human-specific deletion in mouse Cmah increases disease severity in the mdx model of Duchenne muscular dystrophy Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin.

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Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy.

http://www.wikidata.org/entity/Q45883916

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2002 nî lūn-bûn

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Modular flexibility of dystrop ...... f Duchenne muscular dystrophy.

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Modular flexibility of dystrop ...... f Duchenne muscular dystrophy.

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Modular flexibility of dystrop ...... f Duchenne muscular dystrophy.

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Modular flexibility of dystrop ...... f Duchenne muscular dystrophy.

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Modular flexibility of dystrop ...... f Duchenne muscular dystrophy.

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Modular flexibility of dystrop ...... f Duchenne muscular dystrophy.

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Modular flexibility of dystrop ...... f Duchenne muscular dystrophy.

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