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Article

Long-term Skeletal Muscle Protection After Gene Transfer in a Mouse Model of LGMD-2D

24 Jul 2007

Abstract excerpt

Limb girdle muscular dystrophy (LGMD) describes a group of inherited diseases resulting from mutations in genes encoding proteins involved in maintaining skeletal muscle membrane stability. LGMD type-2D is caused by mutations in alpha-sarcoglycan (sgca). Here we describe muscle-specific gene delivery of the human sgca gene into dystrophic muscle using an adeno-associated virus 1 (AAV1) capsid and creatine kinase...

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Identifiers

PMID: 17653106
DOI: 10.1038/sj.mt.6300246

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