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AAV5.Dysf gene delivery. Dysferlin deficient mice (Dysf−/−) mice were treated by intramuscular or vascular delivery via the femoral artery with AAV5.Dysf. Dysferlin expression was restored one month after treatment (right panel) compared to untreated mice (left panel). The overexpression of dysferlin in the muscle fibers is typical of this method of gene transfer and is non-toxic to the cell.
