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• W1415925136 abstract "Publisher Summary This chapter discusses the combination of gene transfer via efficient viral vectors, followed by intracerebral grafting of the genetically modified cells may constitute an effective approach to some disorders of central nervous system (CNS) function. Several prerequisites for a human disease to be suitable as a candidate recipient for transplantation of genetically engineered cells are also discussed, including the pathogenesis and pathophysiology of the disease must be sufficiently well understood for the identification of the relevant gene product to be introduced into defective cells, the relevant gene must be well characterized and available as a clone, or ideally, an animal model should be available. However, the major difficulty with most human diseases of the CNS is that these criteria cannot be satisfied, because the exact mechanisms of pathophysiology and pathogenesis in most human genetic diseases, such as phenylketoneuria and Lesch-Nyhan disease, are not adequately understood. Although, the present models are all very complex, and truly effective therapy is likely to be extremely difficult to achieve, but it seems probable that a number of conceptually new approaches to these previously inaccessible diseases will become feasible, and that such new approaches will include the implantation of genetically modified cells." @default.
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• W1415925136 date "1988-01-01" @default.
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• W1415925136 title "Chapter 86 Implantation of genetically engineered cells to the brain" @default.
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• W1415925136 doi "https://doi.org/10.1016/s0079-6123(08)60345-3" @default.
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