Viral vectors for gene transfer to striated muscle.
Recent progress has generated exciting results that are increasing the prospects for gene therapy of a variety of disorders of striated muscle, including the muscular dystrophies and myopathies, acquired and inherited diseases of cardiac muscle, and aging-associated muscle wasting. Numerous viral vector systems are being employed to transfer therapeutic genes to striated muscles, and advances in vector technology are leading to improved gene transfer efficiencies coupled with reduced immunological responses. The current technology in the field of viral vectors as tools for gene delivery to striated muscle is summarized, and recent developments related to gene therapies for skeletal and cardiac muscle disorders are discussed.
Gregorevic
Paul
P
The University of Washington, Department of Neurology, Senator Paul D Wellstone Muscular Dystrophy Cooperative Research Center, Seattle, WA 98195, USA.
Blankinship
Michael J
MJ
Chamberlain
Jeffrey S
JS
D015316:Genetic Therapy / Q000295:instrumentation* / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics*; D006801:Humans; D018482:Muscle, Skeletal / Q000378:metabolism / Q000473:pathology / Q000821:virology*; D009135:Muscular Diseases / Q000235:genetics* / Q000378:metabolism / Q000473:pathology / Q000628:therapy*; D014780:Viruses / Q000235:genetics*