Dmd – animal models & preclinical treatment

Gene therapy for Duchenne muscular dystrophy (DMD) is being evaluated as a therapeutic strategy to restore production of functional dystrophin. Replacement of full-length dystrophin gene is not feasible due to its large size relative to the cloning capacity of adeno-associated virus (AAV)-derived vectors ( ∼5kb). Strategies based on delivery of shortened, but functional forms of dystrophin have emerged as a viable option. Identification of the optimal shortened micro-dystrophin construct that most normalizes muscle function is a key translation question.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research