GENE-THERAPY OF NEUROLOGICAL DISORDERS - EXPERIMENTAL PARADIGMS AND CLINICAL PERSPECTIVES

So far, it has not been possible to treat many neurological conditions causally. However, in the past few years underlying genetic defects h ave been characterized for a substantial number of neurodegenerative d isorders. Experimental methods have been developed that allow for effi cient gene transfer into defined regions of the mammalian CNS. Such te chniques can be applied to deliver genes into target cells of a recipi ent organism or to transfer genetically modified cells into defined re gions of the CNS. Candidate genes for gene therapy are those encoding for neurotrophins and neurotransmitters for symptomatic therapy and, i n the case of neurodegenerative disorders with localized gene defects, the wild-type allele as a causal treatment approach. In this review a rticle, we describe some of the most widely used strategies for gene t ransfer to the CNS. We also report on the results obtained with animal models for human disease, and discuss both the chances and problems o f gene therapy approaches in clinical medicine.