In situ muscle force assessment and AAV micro-dystrophin gene therapy in a canine model of duchenne

来源 :International Conference for Physiological Sciences 2012(201 | 被引量 : 0次 | 上传用户:qweewqqwe123
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  Duchenne muscular dystrophy (DMD) is a fatal disease caused by dystrophin deficiency.Dystrophy symptom is seen in the canine but not murine DMD models.To test gene therapy efficacy, we developed a novel protocol to transfer adeno-associated virus micro-dystrophin (AAV μdys) vector into the extensor carpi ulnaris muscle (ECU) of dystrophy dogs.We also developed an apparatus to measure the ECU force.Robust and sustained micro-dystrophin expression was achieved following direct AAV μdys injection.
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