Restriction of Calpain3 Expression to the Skeletal Muscle Prevents Cardiac Toxicity and Corrects Pathology in a Murine Model of Limb-Girdle Muscular Dystrophy | Zendy

Carinne Roudaut | Zendy; Florence Le Roy | Zendy; Laurence Suel | Zendy; Jérôme Poupiot | Zendy; Karine Charton | Zendy; Marc Bartoli | Zendy; Isabelle Richard | Zendy

Open Access

Restriction of Calpain3 Expression to the Skeletal Muscle Prevents Cardiac Toxicity and Corrects Pathology in a Murine Model of Limb-Girdle Muscular Dystrophy

Author(s) -

Carinne Roudaut,

Florence Le Roy,

Laurence Suel,

Jérôme Poupiot,

Karine Charton,

Marc Bartoli,

Isabelle Richard

Publication year - 2013

Publication title -

circulation

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 7.795

H-Index - 607

eISSN - 1524-4539

pISSN - 0009-7322

DOI - 10.1161/circulationaha.113.001340

Subject(s) - muscular dystrophy , skeletal muscle , medicine , transgene , cardiac muscle , limb girdle muscular dystrophy , duchenne muscular dystrophy , toxicity , pathology , anatomy , gene , biology , mutation , genetics

Genetic defects in calpain3 (CAPN3) lead to limb-girdle muscular dystrophy type 2A, a disease of the skeletal muscle that affects predominantly the proximal limb muscles. We previously demonstrated the potential of adeno-associated virus-mediated transfer of the CAPN3 gene to correct the pathological signs in a murine model for limb-girdle muscular dystrophy type 2A after intramuscular and locoregional administrations.

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