MYOBLAST TRANSFER IN DUCHENNE MUSCULAR-DYSTROPHY

One biceps muscle of 8 patients with Duchenne muscular dystrophy was i njected at 55 sites with a total of 55 million viable, purified, and c ontamination-free normal myoblasts (myoblast transfer). The other bice ps of each patient was injected with a placebo to serve as a control. The procedure was blinded to the patients, parents, and investigators. Myoblasts derived from a biopsy specimen of the fathers were cultured and purified under strict conditions and carefully screened for micro bial contamination. All patients received cyclophosphamide for immunos uppression for 6 or 12 months. No serious complications were observed after myoblast transfer, indicating that the procedure is safe. The ov erall therapeutic efficiency of myoblast transfer was poor as judged b y the results in maximal voluntary force generation, dystrophin conten t of the muscle, magnetic resonance imaging of the muscle, and the lac k of donor-derived DNA and dystrophin messenger RNA in the injected mu scle. An improved efficiency of the take of myoblasts might be achieve d by using younger cells and injecting the myoblasts with a myonecroti c agent (to increase the prevalence of regeneration) and a basal lamin al fenestrating agent.