Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy.
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Cell membrane expression of cardiac sodium channel Na(v)1.5 is modulated by alpha-actinin-2 interactionFollistatin gene delivery enhances muscle growth and strength in nonhuman primatesRecent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trialsDystrophin and the two related genetic diseases, Duchenne and Becker muscular dystrophiesHomologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transferInhibitory control over Ca(2+) sparks via mechanosensitive channels is disrupted in dystrophin deficient muscle but restored by mini-dystrophin expressionThe Crystal Structures of Dystrophin and Utrophin Spectrin Repeats: Implications for Domain BoundariesAnimal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapyMuscular dystrophy begins early in embryonic development deriving from stem cell loss and disrupted skeletal muscle formationReport 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 TherapyMicroutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient miceSkeletal 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 cellsEnhanced effect of microdystrophin gene transfection by HSV-VP22 mediated intercellular protein transportL-type Ca2+ channel function is linked to dystrophin expression in mammalian muscleSkeletal 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-dystrophinTruncated 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 dystrophyDystrophin 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 vectorsMLPA identification of dystrophin mutations and in silico evaluation of the predicted protein in dystrophinopathy cases from India.Emerging genetic therapies to treat Duchenne muscular dystrophyGene 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 abilityDUX4, a candidate gene for facioscapulohumeral muscular dystrophy, causes p53-dependent myopathy in vivoAdeno-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 membraneRestoration 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 studyA human-specific deletion in mouse Cmah increases disease severity in the mdx model of Duchenne muscular dystrophyFunctional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin.
P2860
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P2860
Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy.
description
2002 nî lūn-bûn
@nan
2002年の論文
@ja
2002年学术文章
@wuu
2002年学术文章
@zh-cn
2002年学术文章
@zh-hans
2002年学术文章
@zh-my
2002年学术文章
@zh-sg
2002年學術文章
@yue
2002年學術文章
@zh
2002年學術文章
@zh-hant
name
Modular flexibility of dystrop ...... f Duchenne muscular dystrophy.
@en
Modular flexibility of dystrop ...... f Duchenne muscular dystrophy.
@nl
type
label
Modular flexibility of dystrop ...... f Duchenne muscular dystrophy.
@en
Modular flexibility of dystrop ...... f Duchenne muscular dystrophy.
@nl
prefLabel
Modular flexibility of dystrop ...... f Duchenne muscular dystrophy.
@en
Modular flexibility of dystrop ...... f Duchenne muscular dystrophy.
@nl
P2093
P356
P1433
P1476
Modular flexibility of dystrop ...... f Duchenne muscular dystrophy.
@en
P2093
Ann S Robinson
Christiana DelloRusso
Hollie A Harper
Jeffrey S Chamberlain
John F Engelhardt
Michael A Hauser
Robert W Crawford
Scott Q Harper
Stephanie F Phelps
Susan V Brooks
P2888
P304
P356
10.1038/NM0302-253
P407
P577
2002-03-01T00:00:00Z
P5875
P6179
1007037005