Gene therapy in neurology

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Abstract
Many methods of gene transfer to the brain are under study for the treatment of the central nervous system manifestations of a number of diseases. One strategy employs vectors--typically genetically altered viruses--for the delivery of exogenous genes directly to a host's brain cells in situ. Alternatively, transplanting cells--of either neural or nonneural origin--that intrinsically secrete missing or therapeutic gene produces, or which are genetically engineered ex vivo to do so, may provide another strategy. Nonmigratory cells implanted into small, discrete anatomic sites are well suited for disease processes whose pathology is very focal. Neural cells that have the capacity to migrate in the central nervous system provide more powerful vehicles for delivering factors to disease involving pathology that is widely disseminated, extensive, multifocal, or even global (e.g., many neurogenetic diseases). Both neural and nonneural cell types have been successfully engineered to produce a variety of neurotransmitter synthetic enzymes, metabolic enzymes, and neurotrophic factors. Impressive results with genetically modified cells in animal models of central nervous system disease encourage the continued development of gene therapy strategies for treating neural dysfunction.

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