Recent advances in skeletal-muscle-based gene therapy.
Gene transfer into skeletal muscle holds promise for the treatment of a variety of serum protein deficiencies, muscular dystrophies, and chronic ischemic limb syndromes. The past two years have seen the development of new and improved vectors for programming recombinant gene expression in skeletal muscle. Important advances include first, novel plasmid DNA, adenovirus, and adeno-associated virus vectors that can be used to stably express therapeutic levels of recombinant proteins in the skeletal muscle of immunocompetent hosts and second, the development of vector systems that enable regulated and tissue-specific transgene expression in skeletal muscle in vivo.
Department of Medicine, University of Chicago, Illinois 60637, USA.
D J
DJ
Marshall
J M
JM
Leiden
D000818:Animals; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000235:genetics; D006801:Humans; D018482:Muscle, Skeletal / Q000166:cytology / Q000378:metabolism*