Expression of dystrophin in Duchenne muscular dystrophy patients after myoblast transplantation

Based on originally designed technique of myoblast cultivation and in accor dance with the approved by the Russian Ministry of Health "one muscle treat ment" protocol of myoblast transplantation to the Duchenne muscular dystrop hy patients, the first in Russia clinical trial of this gene correction met hod was carried out. Immunologically related myoblast cultures (30 to 90 mi llion cells per patient) were injected after all preliminary procedures int o tibialis anterior muscles of four boys selected from a group of volunteer recipients (Duchenne muscular dystrophy patients) based on the analysis of a number of surface antigens in donor-recipient pairs. The condition of th e patients remained satisfactory during the whole period of post-transplant ation follow-up (from 6 months to 1.5 years). Six months after myoblast tra nsplantation the presence of donor DNA or dystrophin synthesis was demonstr ated in muscle biopsies of three out of four patients. This result confirms efficacy and safety of the procedure used.