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Article

AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Nov 2011

Bortolanza Sergia, Nonis Alessandro, Sanvito Francesca, Maciotta Simona, Sitia Giovanni, Wei Jessica, Torrente Yvan, Di Serio Clelia, Chamberlain Joel R, Gabellini Davide

Abstract excerpt

Treatment of dominantly inherited muscle disorders remains a difficult task considering the need to eliminate the pathogenic gene product in a body-wide fashion. We show here that it is possible to reverse dominant muscle disease in a mouse model of facioscapulohumeral muscular dystrophy (FSHD)....

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Share to Dependovirus
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Injections, Intravenous

Share to Microfilament Proteins

Share to Muscular Dystrophy, Facioscapulohumeral

Share to RNA-Binding Proteins

Share to RNA, Small Interfering

Share to Transduction, Genetic

Source

Open in PubMed Open full text

Identifiers

PMID: 21829175
DOI: 10.1038/mt.2011.153
PMCID: PMC3222524

Genes

FRG1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-21829175-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

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