Transplantation of adult-derived myoblasts in mice following gene transfer

We have explored the use of myoblasts obtained from adult animals as a target for somatic gene therapy. Myoblasts from an adult beta-glucuronidase deficient (MPS VII) mouse were isolated and infected with a retroviral vector carrying the human beta-glucuronidase cDNA. Beta-glucuronidase was used as a reporter gene to follow the fate of genetically-modified myoblasts after transplantation into the tibialis anterior of MPS VII recipients. When experimental necrosis had been induced in the recipient muscle prior to cell injection, histological analysis demonstrated efficient engraftment of adult derived myoblasts following gene transfer. The reconstituted myofibres expressed the transgene for at least 10 weeks following transplantation.