Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
about
Restoration of full-length SMN promoted by adenoviral vectors expressing RNA antisense oligonucleotides embedded in U7 snRNAsHypernitrosylated ryanodine receptor calcium release channels are leaky in dystrophic musclemiR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapyEnhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapyAntisense-mediated exon skipping: a versatile tool with therapeutic and research applicationsDystrophin and the two related genetic diseases, Duchenne and Becker muscular dystrophiesNanoparticle delivery of antisense oligonucleotides and their application in the exon skipping strategy for Duchenne muscular dystrophySplicing therapy for neuromuscular diseasePerspectives of stem cell therapy in Duchenne muscular dystrophyGene therapy targets in heart failure: the path to translationAnimal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapyU7 snRNAs: a computational surveyDoxycycline-controlled splicing modulation by regulated antisense U7 snRNA expression cassettesRescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in miceMYBPC3 in hypertrophic cardiomyopathy: from mutation identification to RNA-based correctionAAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasisThe mdx mouse model as a surrogate for Duchenne muscular dystrophyRNA and diseaseThe alternative heart: impact of alternative splicing in heart disease.DHPR alpha1S subunit controls skeletal muscle mass and morphogenesis.Amelioration of Duchenne muscular dystrophy in mdx mice by elimination of matrix-associated fibrin-driven inflammation coupled to the αMβ2 leukocyte integrin receptorEnhancing muscle membrane repair by gene delivery of MG53 ameliorates muscular dystrophy and heart failure in δ-Sarcoglycan-deficient hamsters.Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skippingBlastocyst injection of wild type embryonic stem cells induces global corrections in mdx mice.Functional substitution by TAT-utrophin in dystrophin-deficient mice.Exon exchange approach to repair Duchenne dystrophin transcriptsDelivery is key: lessons learnt from developing splice-switching antisense therapiesTherapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.Role of cyclic AMP-dependent kinase response element-binding protein in recombinant adeno-associated virus-mediated transduction of heart muscle cells.Producing recombinant adeno-associated virus in foster cells: overcoming production limitations using a baculovirus-insect cell expression strategy.Gene-mediated restoration of normal myofiber elasticity in dystrophic muscles.Influence of immune responses in gene/stem cell therapies for muscular dystrophies.Gene and cell-mediated therapies for muscular dystrophy.Differential requirement for utrophin in the induced pluripotent stem cell correction of muscle versus fat in muscular dystrophy mice.Combination of myostatin pathway interference and dystrophin rescue enhances tetanic and specific force in dystrophic mdx micePeripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.Dystrophins, utrophins, and associated scaffolding complexes: role in mammalian brain and implications for therapeutic strategies.Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.A human-specific deletion in mouse Cmah increases disease severity in the mdx model of Duchenne muscular dystrophyTranslation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.
P2860
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P2860
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
description
2004 nî lūn-bûn
@nan
2004 թուականի Դեկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2004 թվականի դեկտեմբերին հրատարակված գիտական հոդված
@hy
2004年の論文
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2004年学术文章
@wuu
2004年学术文章
@zh-cn
2004年学术文章
@zh-hans
2004年学术文章
@zh-my
2004年学术文章
@zh-sg
2004年學術文章
@yue
name
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
@ast
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
@en
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
@nl
type
label
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
@ast
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
@en
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
@nl
prefLabel
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
@ast
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
@en
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
@nl
P2093
P3181
P356
P1433
P1476
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
@en
P2093
Adeline Vulin
France Leturcq
Françoise Fougerousse
Jean-Claude Kaplan
Luis Garcia
Olivier Danos
P304
P3181
P356
10.1126/SCIENCE.1104297
P407
P577
2004-11-04T00:00:00Z