Adenovirus-mediated gene delivery to skeletal muscle.

Adenoviral vectors can be employed for gene delivery to skeletal muscle, both ex vivo and in vivo. Although the realization of the full potential of adenoviral vectors awaits the development of methods to allow safe and efficient targeted gene delivery to mature skeletal muscle upon intravenous vector administration (1), the current generation of vectors has nonetheless found utility in preclinical studies of gene therapy and in gene-transfer experiments designed to study muscle biology. Features of adenoviral vectors that have favored their use for gene delivery to skeletal muscle include the ability to infect both actively dividing and terminally differentiated cells, as well as their large insert capacity. Gutted adenoviral vectors are capable of carrying the large dystrophin gene together with regulatory sequences, and are therefore appropriate vehicles for gene-replacement therapy for Duchenne muscular dystrophy. In addition to their suitability for in vivo gene-therapy applications, adenoviral vectors have been used ex vivo to transfer genes to myoblasts prior to myoblast transplantation into muscle.