Lack of mRNA and dystrophin expression in DMD patients three months after myoblast transfer.
We report our experience on myoblast transplantation in three Duchenne muscular dystrophy patients. Pure myoblasts (55 x 10(6) per patient) from HLA-matched donors, were injected into a tibialis anterior and the controlateral muscle was sham injected. Three months after transplantation, biopsies from the injected muscles were negative for dystrophin expression by immunocytochemistry. Reverse transcriptase-PCR (RT-PCR) failed to amplify any fragments of the deleted regions. This result confirms that myoblast transplantation is feasible, although the efficacy of this therapeutic approach is poor.
Morandi
L
L
Department of Neuromuscular Diseases, Istituto Nazionale Neurologico C. Besta, Milan, Italy.
Bernasconi
P
P
Gebbia
M
M
Mora
M
M
Crosti
F
F
Mantegazza
R
R
Cornelio
F
F
D000328:Adult; D001483:Base Sequence; D001706:Biopsy; D015153:Blotting, Western; D002648:Child; D004247:DNA / Q000032:analysis; D016189:Dystrophin / Q000032:analysis* / Q000235:genetics; D006801:Humans; D007273:Injections, Intramuscular; D008297:Male; D008969:Molecular Sequence Data; D018485:Muscle Fibers, Skeletal / Q000637:transplantation*; D009136:Muscular Dystrophies / Q000628:therapy*; D016133:Polymerase Chain Reaction; D012333:RNA, Messenger / Q000032:analysis; D013997:Time Factors
D016189:Dystrophin; D012333:RNA, Messenger; D004247:DNA