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Article

Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model

16 Jan 2023

Abstract excerpt

Limb-girdle muscular dystrophy (LGMD) type 2C/R5 results from mutations in the γ-sarcoglycan ( SGCG ) gene and is characterized by muscle weakness and progressive wasting. Loss of functional γ-sarcoglycan protein in the dystrophin-associated protein complex destabilizes the sarcolemma, leading to eventual myofiber death. The SGCG knockout mouse ( SGCG −/− ) has clinical-pathological features that replicate the...

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Identifiers

PMID: 36816759
DOI: 10.1016/j.omtm.2023.01.004

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