GENE-THERAPY FOR DISEASES OF THE NERVOUS-SYSTEM

Advances in molecular biology and recombinant DNA technologies have co ntributed to our understanding of the molecular basis of many diseases . Now the possibility of gene transfer into normal cells to produce a gene product of therapeutic potential, or into diseased cells to corre ct the pathologic alteration, promises to revolutionize medical practi ce. In contemporary medicine, many therapeutic strategies focus on the link between a biochemical deficiency and the ensuing disorder. The t reatment of noninfectious disease is often based on replacement therap y; medication is given to compensate for biochemical defects and to pr event or reverse the progression of disease. Although conventional the rapies seldom alter the fundamental cause of a disease, gene therapy p otentially could correct, at a molecular level, the genetic abnormalit ies contributing to its pathogenesis. Treatment directed at specific m olecular alterations associated with the development of neurologic dis ease provides expectations of more effective and less toxic therapy. T he development of gene therapy for nervous system tumors has progresse d rapidly and may be prototypical in the development of therapies for inherited and acquired disorders of the nervous system. We describe po ssible strategies for using gene therapy to treat nervous system disor ders, and we review recent advances in gene therapy for nervous system tumors.