Human Myoblast Genome Therapies and Devices in Regenerative Medicine

This article reviews the scientific and intellectual property development of a biotechnology platform in regenerative medicine called Human Myoblast Genome Therapy (HMGT), known previously as Myoblast Transfer Therapy (MTT). Myoblasts are the least differentiated myogenic cells capable of extensive division, natural cell fusion, nucleus transfer, cell therapy and genome therapy. Myoblasts cultured from muscle biopsy survive, develop and function, after transplantation in animal studies and clinical trials, to revitalize degenerative organs in heart failure, ischemic cardiomyopathy, Type II diabetes, muscular dystrophies, aging dysfunction and disfigurement. Myoblasts have also been used to enhance skin and muscle appearance in cosmetology. HMGT replenishes live cells and genetically repairs degenerating myofibers. It is the world's first human gene therapy when it replenished dystrophin in Duchenne muscular dystrophy as reported in Lancet on July 14, 1990. Data from FDA- approved Phase II/III muscular dystrophy clinical trials demonstrated significant safety and efficacy to merit allowance of cost recovery in consecutive years. Data from FDA- and EMA- approved Phase II/III ischemic cardiomyopathy clinical trials demonstrated significant safety and efficacy. This review also provides in-depth analyses of key factors related to success and failure of HMGT procedures. Future development will focus on myoblasts transduced with VEGF165 using nanoparticles or liposomes that are promising biologics for angiomyogenesis. Automated cell processors, myogenic cell injection catheters and methods of use have been patented to complement the HMGT technology.