Gene Therapy for Human Genetic Disease?

摘要: In our view, gene therapy may ameliorate some human genetic diseases in the future. For this reason, we believe that research directed at development of techniques for should continue. foreseeable future, however, oppose any further attempts patients because (i) understanding such basic processes as regulation and recombination cells is inadequate; (ii) details relation between molecular defect disease state rudimentary essentially all diseases; (iii) have no information on short-range long-term side effects therapy. We therefore propose a sustained effort be made to formulate complete set ethicoscientific criteria guide clinical application techniques. Such an endeavor could go long way toward ensuring used humans only those instances where it will prove beneficial, preventing its misuse through premature application. Two recent papers provided new demonstrations modification mammalian cells. Munyon et al. (44) restored ability synthesize enzyme thymidine kinase kinase-deficient mouse by infection with ultraviolet-irradiated herpes simplex virus. their experiments DNA from virus, which contains coding kinase, formed hereditable association Merril (45) reported treatment fibroblasts galactosemia exogenous caused increased activity missing enzyme, α-D-galactose-l-phosphate uridyltransferase. They also evidence change persisted after subculturing treated If latter report can confirmed, feasibility would clearly demonstrated, considerably enhancing technical prospects