Pioneering Human Myoblast Genome Therapy

Human Myoblast Genome Therapy (HMGT) is a platform technology of cell transplantation, nuclear transfer, and tissue engineering. Myoblasts are differentiated, immature cells destined to become muscles. Myoblasts cultured from muscle biopsy survive, develop and function to revitalise degenerative muscles upon transplantation. Transplant injury activates regeneration of host myofibers that fuse with the injected myoblasts, sharing their nuclei in a common gene pool of the syncytium. Thus, through nuclear transfer and complementation, human genome can be transferred into muscles of genetically-ill patients to achieve phenotype repair. Myoblasts are safe and efficient universal gene transfer vehicles endogenous to muscles that constitute 50% of the body. Myoblasts fuse among themselves to form new myofibres. Patients take only 2-month cyclosporine to immunosuppress allograft rejection because myofibres do not express MHC-1 antigens. The first correction of human gene defect was published in the Lancet on July 14, 1990 when the therapeutic protein dystrophin was found in the myoblast-injected muscle of a Duchenne muscular dystrophy (DMD) patient. Results over 280 HMGT procedures on MD subjects in the past 15 years demonstrated absolute safety. Myoblast-injected DMD muscles showed improved histology. Strength increase at 18 months post-operatively averaged 123%. FDA-approved clinical trials progressed unto Phase III in USA with direct cost recovery. Heart muscle degeneration is the leading cause of human debilitation and death.