Human Myoblast Genome Therapies and Devices in Regenerative Medicine (2011)

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 worlds 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. Keywords: Myoblasts, biologics, muscular dystrophies, heart failure, ischemic cardiomyopathy, Type II diabetes, anti-aging cosmetics, automated cell processors, catheters, Human Myoblast Genome Therapy (HMGT), Myoblast Transfer Therapy (MTT), transplantation techniques, myogenic cells, biopsy, Duchenne muscular dystrophy (DMD), hereditary degenerative diseases, replenish live cells, Cell fusion, Genome therapy, multi-nucleated heterokaryon, vascular endothelial growth factor (VEGF165), Allograft immunogenicity, current good manufacture practices (cGMP), standard operation procedures (SOP's), Drug Master File (DMF), mycoplasmal contamination, penicillin, myoblast injection techniques, SKELETAL MUSCLE REGENERATION, dystrophic satellite cells, Mesenchymal tissue, cyclosporine-A, dystrophin, immunocytochemical localization, horseradish peroxidase (HRP), immunocytochemistry, Dulbecco's Modified Eagles Medium (DMEM), phosphatebuffered saline (PBS), sarcolemma, gel electrophoresis, Histoincompatible Transplants, Cyclosporine, Histocompatible Transplants, muscle twitch tension, tetanus tension, mechanophysiology, supramaximal nerve stimulation, Sporadic flexion, flaccid extension, hybrid isozymes, European Patent Agency (EPA), Myoblast Therapy for Mammalian Diseases, somatic cell therapies, gene therapies, double-blind study, phagocytic necrosis, sham-injected muscles, first human gene therapy, lower body treatment (LBT), ankle plantar flexors, knee flexors, knee extensors, ambulatory subjects, degenerated proximal muscles, myogenicity, neuromuscular junctions, T-lymphocyte proliferation, whole body treatment (WBT), infantile facioscapulohumeral dystrophy, isometric force, creatine kinase (CK), aspartate aminotransferase, Duchenne Muscular Dystrophy, HEART MUSCLE DEGENERATION, congestive heart failure, endomyocardial injections, electrocardiogram, Transmyocardial perforation, Myostar catheter, regenerative heart, Single-Photon Emission Computed Tomography, multinucleated heterokaryons, xenografts, reverse transcription polymerase chain reaction, angiogenesis, Polyethylenimine-25 nanoparticles, coronary artery bypass grafting (CABG), atherosclerosis, Echocardiography, fibroblast infiltration, ANTI-AGING AESTHETICA (AAA), Regenerative Medicine, Cell transplantation, Angiomyogenesis, cytogenetics