Viral and non-viral methods for gene transfer into skeletal muscle.
Skeletal muscle is an important target for genetic manipulation and its stable post-mitotic nature allows the use of both integrating and non-integrating viral and non-viral vectors. Adeno-associated viral vectors and naked plasmid DNA are currently the vectors of choice for gene transfer into muscle. The last couple of years have seen major breakthroughs in the field of vector delivery systems, particularly those using the vascular route, such that gene therapy of muscular dystrophies and the use of muscle as a platform for the production of secreted proteins has become a clinical possibility.
Wells
Dominic J
DJ
Imperial College London, Department of Cellular and Molecular Neuroscience, Division of Neuroscience and Mental Health, Charing Cross Hospital, UK. d.wells@imperial.ac.uk
D000818:Animals; D004247:DNA / Q000008:administration & dosage / Q000627:therapeutic use; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D006467:Hemophilia A / Q000235:genetics / Q000628:therapy; D006801:Humans; D018482:Muscle, Skeletal / Q000502:physiology*; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000628:therapy; D014780:Viruses / Q000235:genetics*
D004247:DNA