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Myoblast Transfer in the Tratment of Duchenne's Muscular Dystrophy
NEJM 333:832-838, 8711995., Mendell,J.R.,et al, 1995
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Article Abstract
There was no significant difference in muscle strength between arms injected with myoblasts and sham-injected arms.In one patient,10.3 percent of muscle fibers expressed donor-derived dystrophin after myoblast transfer.Three other patients also had a low level of donor dystrophin(<1 percent);eight had none.Myoblasts transferred once a month for six months failed to improve strength in patients with Duchenne's muscular dystrophy. The value of exon-specific peptide antibodies in the interpretation of myoblast-transfer results was demonstrated in a patient with Duchenne's muscular dystrophy who had a high percentage of donor-derived dystrophin. Specific variables affecting the efficiency of myoblast transfer need to be identified in order to improve upon this technique.
 
Related Tags
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dystrophin
gene therapy
muscular dystrophy
muscular dystrophy,Duchenne
myoblast transfer
treatment of neurologic disorder

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