GENE-THERAPY FOR NEUROLOGIC DISEASE - BENCHTOP DISCOVERIES TO BEDSIDEAPPLICATIONS .1. THE BENCH

The overall goal of this review is to provide the pediatric neurologis t with a theoretical foundation in gene therapy. Gene therapy became f easible in the early 1970s and the first transfer of a foreign gene in to humans was approved by the NM in 1989. Adenovirus, adeno-associated virus, herpes-simplex virus, retroviruses, and other vectors have bee n used to efficiently transduce genes into cells in vitro and in vivo. We discuss laboratory experiments that have provided a strong scienti fic rationale for implementing human clinical trials of gene therapy f or neurologic malignancy. The development of viral and nonviral vector s that mediate efficient gene insertion into human cells has created t he prospect of using gene therapy for cancer or brain disease. The NM has approved more than 100 gene therapy protocols since 1989. However, the field will require more research on gene delivery systems before gene therapy becomes an established therapeutic strategy for an array of central nervous system diseases.