Gene delivery to dystrophic muscle.
Electroporation is a powerful method for gene delivery to dystrophic muscle in the mdx mouse model of Duchenne muscular dystrophy. Successful transfer of reporter and therapeutic plasmids and antisense oligonucleotides has been demonstrated. However, the efficiency falls with increasing plasmid size. Although it is unlikely that the electrotransfer approach will be useful clinically, it is an important experimental tool, particularly in testing potential immune responses to gene transfer in the absence of vector proteins.
Wells
Kim E
KE
Department of Cellular and Molecular Neuroscience, Division of Neuroscience and Mental Health, Imperial College, London, United Kingdom.
McMahon
Jill
J
Foster
Helen
H
Ferrer
Aurora
A
Wells
Dominic J
DJ
D000818:Animals; D004274:DNA, Recombinant / Q000008:administration & dosage / Q000235:genetics; D016189:Dystrophin / Q000235:genetics / Q000378:metabolism; D053672:Electrochemotherapy / Q000379:methods*; D015870:Gene Expression; D017930:Genes, Reporter; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D007150:Immunohistochemistry; D007273:Injections, Intramuscular; D051379:Mice; D018101:Mice, Inbred mdx; D018482:Muscle, Skeletal / Q000378:metabolism; D009137:Muscular Dystrophy, Animal / Q000235:genetics / Q000378:metabolism / Q000628:therapy*; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000628:therapy; D010957:Plasmids / Q000008:administration & dosage / Q000235:genetics; D001616:beta-Galactosidase / Q000235:genetics / Q000378:metabolism
D004274:DNA, Recombinant; D016189:Dystrophin; D001616:beta-Galactosidase