Pilot study of myoblast transfer in the treatment of Becker muscular dystrophy.
We evaluated myoblast implantation therapy in three subjects with Becker muscular dystrophy who received 60 million myoblasts in one tibialis anterior (TA) muscle 2 months after beginning cyclosporine immunosuppression (5 to 10 mg/kg) that continued for 1 year. Strength of the implanted and control TA muscles was measured before and after treatment using a gauge to record TA contraction force. Our protocol controlled for the effects of cyclosporine and myoblast injections. In this pilot study, myoblast implantation did not improve strength of the implanted TA muscles.
Neumeyer
A M
AM
Department of Neurology, Massachusetts General Hospital, Boston 02129, USA.
Cros
D
D
McKenna-Yasek
D
D
Zawadzka
A
A
Hoffman
E P
EP
Pegoraro
E
E
Hunter
R G
RG
Munsat
T L
TL
Brown
R H
RH
D000328:Adult; D002478:Cells, Cultured; D016572:Cyclosporine / Q000627:therapeutic use; D004311:Double-Blind Method; D016189:Dystrophin / Q000235:genetics; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D009132:Muscles / Q000166:cytology / Q000637:transplantation*; D009136:Muscular Dystrophies / Q000628:therapy*; D010865:Pilot Projects; D015203:Reproducibility of Results; D012680:Sensitivity and Specificity; D033581:Stem Cell Transplantation; D014019:Tissue Donors
D016189:Dystrophin; D007166:Immunosuppressive Agents; D016572:Cyclosporine