Chapter 17 Gene therapy in the CNS: Intracerebral grafting of genetically modified cells

摘要: Grafting cells to the CNS has been suggested and applied as a potential approach therapy through selective replacement of lost result disease or damage. Independently, studies aimed at direct genetic in model systems have recently begun suggest conceptually new approaches treatment several kinds human disease, especially those caused by single gene enzyme deficiencies. We that combination these two approaches, namely graftment into genetically modified cells, may provide toward restoration some functions damaged diseased CNS. present evidence for feasibility this approach, including description current techniques mammalian cell transfer grafting, possible clinical applications. Specifically, we report fibroblasts, secrete NGF infection with retroviral vector implanted brains rats surgical lesion fimbria-fornix, prevented degeneration cholinergic neurons would die without treatment.