Cell transplantation and gene therapy in muscular dystrophy.
Duchenne's muscular dystrophy (DMD), which affects 1/3500 live male births, involves a progressive degeneration of skeletal and cardiac muscle, leading to early death. The protein dystrophin is lacking in DMD and present, but defective, in the allelic, less severe, Becker muscular dystrophy and is also missing in the mdx mouse. Experiments on the mdx mouse have suggested two possible therapies for these myopathies. Implantation of normal muscle precursor cells (mpc) into mdx skeletal muscle leads to the conversion of dystrophin-negative fibres to -positive, with consequent improvement in muscle histology. Direct injection of dystrophin cDNA into skeletal or cardiac muscle also gives rise to dystrophin-positive fibres. Although both appear promising, there are a number of questions to be answered and refinements to be made before either technique could be considered possible as treatments for myopathies in man.
Department of Histopathology, Charing Cross and Westminster Medical School, London, UK.
J E
JE
Morgan
T A
TA
Partridge
D000818:Animals; D016572:Cyclosporine / Q000627:therapeutic use; D018076:DNA, Complementary / Q000008:administration & dosage / Q000493:pharmacokinetics / Q000627:therapeutic use*; D016189:Dystrophin / Q000172:deficiency / Q000235:genetics*; D015316:Genetic Therapy; D006084:Graft Rejection / Q000517:prevention & control; D007273:Injections, Intramuscular; D051379:Mice; D018101:Mice, Inbred mdx; D008819:Mice, Nude; D008845:Microinjections; D009132:Muscles / Q000276:immunology / Q000473:pathology; D009136:Muscular Dystrophies / Q000628:therapy*; D009137:Muscular Dystrophy, Animal / Q000473:pathology / Q000628:therapy*; D051381:Rats; D033581:Stem Cell Transplantation
D018076:DNA, Complementary; D016189:Dystrophin; D016572:Cyclosporine