Retroviral-mediated transfer of a dystrophin minigene into mdx mouse myoblasts in vitro.
We have demonstrated expression of a 6.3 kb Becker muscular dystrophy (BMD) human dystrophin cDNA following retroviral-mediated transduction of cultured myoblasts from the dystrophin-deficient mdx mouse. The truncated dystrophin protein was localised to the sarcolemma of differentiated myotubes by antibodies against the C-terminus of the molecule, and produced an identical immunostaining pattern to that observed in control myotubes expressing normal endogenous dystrophin. These results indicate that retroviral-mediated gene transfer may be useful for experimental in vivo studies on the complementation of dystrophin gene mutations.
Dunckley
M G
MG
Department of Experimental Pathology, UMDS, Guy's Hospital, London, UK.
Love
D R
DR
Davies
K E
KE
Walsh
F S
FS
Morris
G E
GE
Dickson
G
G
D000818:Animals; D002478:Cells, Cultured; D016189:Dystrophin / Q000235:genetics*; D015870:Gene Expression; D005822:Genetic Vectors / Q000235:genetics; D051379:Mice; D008817:Mice, Mutant Strains; D009132:Muscles / Q000166:cytology; D009136:Muscular Dystrophies / Q000235:genetics*; D010957:Plasmids / Q000235:genetics; D012190:Retroviridae / Q000235:genetics; D012508:Sarcolemma / Q000378:metabolism; D014162:Transfection
D016189:Dystrophin