DNA-Mediated Gene Therapy in a Mouse Model of Limb Girdle Muscular Dystrophy 2B

DNA-Mediated Gene Therapy in a Mouse Model of Limb Girdle Muscular Dystrophy 2B

Mutations in the gene for dysferlin cause a degenerative disorder of skeletal muscle known as limb girdle muscular dystrophy 2B. To achieve gene delivery of plasmids encoding dysferlin to hind limb muscles of dysferlin knockout mice, we used a vascular injection method that perfused naked plasmid DNA into all major muscle groups of the hind limb. We monitored delivery by luciferase live imaging...

Limb-girdle Muscular Dystrophy

Limb-girdle Muscular Dystrophy

Limb-girdle Muscular Dystrophy

Limb-girdle muscular dystrophy type 2B mimicking polymyositis.

Dr. Francisco de Assis Aquino Gondim – Universidade Federal do Ceará / CP 3157 Rua Cel Nunes de Melo 1127 60430-270 Fortaleza CE Brasil. E-mail: [email protected] Limb-girdle muscular dystrophy (LGMD) type 2B is caused by mutations in the dysferlin gene, located at cromossome 2p13.1-p13.3. This protein is normally expressed at the skeletal muscle, but in affected patients it is reduced or a...