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Article

Phenotypic correction of alpha-sarcoglycan deficiency by intra-arterial injection of a muscle-specific serotype 1 rAAV vector.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Jan 2007

Fougerousse Francoise, Bartoli Marc, Poupiot Jérôme, Arandel Ludovic, Durand Muriel, Guerchet Nicolas, Gicquel Evelyne, Danos Olivier, Richard Isabelle

Abstract excerpt

alpha-Sarcoglycanopathy (limb-girdle muscular dystrophy type 2D, LGMD2D) is a recessive muscular disorder caused by deficiency in alpha-sarcoglycan, a transmembrane protein part of the dystrophin-associated complex. To date, no treatment exists for this disease. We constructed recombinant pseudotype-1 adeno-associated virus (rAAV) vectors expressing the human alpha-sarcoglycan cDNA from a ubiquitous or a...

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Identifiers

PMID: 17164775
DOI: 10.1038/sj.mt.6300022

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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