Key result
AAV-mediated sgca gene delivery restores muscle mechanics and provides long-term protection in mice.
Why the study?
Does AAV1-mediated sgca gene transfer improve muscle protection and mechanical properties in a mouse model of LGMD-2D?
Population
Adult sgca(-/-) mice (mouse model of Limb girdle muscular dystrophy type-2D)
Comparison
Muscle-specific gene delivery of the human sgca… vs Untreated controls
Design
Preclinical
Authors
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Supports preclinical AAV gene therapy development for LGMD-2D; leaves open human translation.
Does AAV1-mediated sgca gene transfer improve muscle protection and mechanical properties in a mouse model of LGMD-2D?
AAV1-mediated sgca gene transfer provides long-term muscle protection and restores mechanical properties in a mouse model of LGMD-2D.
Pacak et al. (2007) studied Limb girdle muscular dystrophy type-2D (LGMD-2D). AAV-mediated human sgca gene delivery vs. Untreated controls was evaluated on Muscle mechanical properties and disease progression. AAV-mediated delivery of the human sgca gene in adult sgca(-/-) mice restored normal muscle mechanical properties and provided long-term muscle protection compared with untreated controls.
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