TOWARDS GENE-THERAPY FOR THE CENTRAL-NERVOUS-SYSTEM

Gene therapy has generated enormous scientific, medical and public int erest over the last decade. Clinical trials involving approximately 20 00 patients worldwide have targeted simple genetic diseases such as cy stic fibrosis, muscular dystrophy, adenosine deaminase deficiency, Gau cher's disease and familial hypercholesterolemia, as well as complex a cquired diseases such as cancer and AIDS. The central nervous system i s a new and particularly exciting target for gene therapy because its unique properties prevent the successful treatment of many neurologica l disorders by conventional means. This review discusses the potential applications of in vivo gene therapy to neurological disorders that h ave the greatest potential for genetic treatments.