Progress, problems, and prospects for gene therapy in muscle.

Abstract

As the molecular defects that cause many muscle diseases have been identified, research has shifted to finding novel therapies. Gene therapy has been proposed both for correcting primary gene defects of muscle and as a way of using muscle for the production of proteins therapeutic in inflammatory or nonmuscle diseases. Several strategies have been developed to introduce foreign genes into diseased muscles, including myoblast transfer, direct injection of plasmids or DNA-liposome complexes, and infection with modified viruses. Related strategies, using antisense sequences or ribozymes, have been devised to modify gene expression in diseased cells. No method has yet proved itself in the clinic, although current work remains promising and some of the pitfalls that must be overcome have been identified.