Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping - sprookjes - yvandenbroek - TriplyDB

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

http://www.wikidata.org/entity/Q28291830

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Restoration of full-length SMN promoted by adenoviral vectors expressing RNA antisense oligonucleotides embedded in U7 snRNAs Hypernitrosylated ryanodine receptor calcium release channels are leaky in dystrophic muscle miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications Dystrophin and the two related genetic diseases, Duchenne and Becker muscular dystrophies Nanoparticle delivery of antisense oligonucleotides and their application in the exon skipping strategy for Duchenne muscular dystrophy Splicing therapy for neuromuscular disease Perspectives of stem cell therapy in Duchenne muscular dystrophy Gene therapy targets in heart failure: the path to translation Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy U7 snRNAs: a computational survey Doxycycline-controlled splicing modulation by regulated antisense U7 snRNA expression cassettes Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice MYBPC3 in hypertrophic cardiomyopathy: from mutation identification to RNA-based correction AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis The mdx mouse model as a surrogate for Duchenne muscular dystrophy RNA and disease The 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 receptor Enhancing 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 skipping Blastocyst 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 transcripts Delivery is key: lessons learnt from developing splice-switching antisense therapies Therapy 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 mice Peripheral 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 dystrophy Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.

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P2860

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

http://www.wikidata.org/entity/Q28291830

description

2004 nî lūn-bûn

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2004 թուականի Դեկտեմբերին հրատարակուած գիտական յօդուած

@hyw

2004 թվականի դեկտեմբերին հրատարակված գիտական հոդված

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2004年の論文

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2004年学术文章

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2004年学术文章

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2004年学术文章

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2004年学术文章

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2004年学术文章

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2004年學術文章

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name

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

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Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

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Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

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type

label

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

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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

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Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

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Adeline Vulin

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France Leturcq

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Françoise Fougerousse

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Jean-Claude Kaplan

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Luis Garcia

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Olivier Danos

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Aurélie Goyenvalle

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