Books > Medicine > Pre-clinical medicine: basic sciences > Medical genetics
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Pioneering Human Myoblast Genome Therapy (Paperback)
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Pioneering Human Myoblast Genome Therapy (Paperback)
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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.
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