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Gene therapy and the brain.

H A Jinnah·T Friedmann
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Abstract

In this article we describe the application of the emerging concepts of gene therapy to 4 different neurologic disorders. The first of these is Lesch-Nyhan disease, a genetically-determined neurodevelopmental disorder caused by a defect in the gene which encodes the purine salvage enzyme hypoxanthine-guanine phosphoribosyltransferase (HPRT). Two additional disorders, Parkinsonism and Alzheimer's disease, are both neurodegenerative diseases of unknown etiology which affect the elderly. The final disorder involves malignant brain tumors. In each of these disorders, basic research with in vitro systems and animal models has suggested that the tools of gene transfer may provide a novel and potentially effective treatment strategy.

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Alzheimer DiseaseAnimalsBrain NeoplasmsGene Transfer TechniquesGenetic TherapyGenetic VectorsHumansInfant, NewbornLesch-Nyhan SyndromeParkinson DiseaseRatsJournal ArticleReview

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BibTeXRIS

H A Jinnah, T Friedmann. 1995. Gene therapy and the brain.. https://doi.org/10.1093/oxfordjournals.bmb.a072943

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