Pioneering Human Myoblast Genome Therapy by Peter K. Law (English) Paperback Boo
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. 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.
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