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Topical Review: Gene Therapy for Neurologic Disease: Benchtop Discoveries to Bedside Applications. 1. The Bench

Neuro-Oncology Program and the Departments of Pediatrics, University of Florida Brain Institute and Cancer Center, Gainesville, FL, Department of Neurology, University of Florida Brain Institute and Cancer Center, Gainesville, FL, Department of Neuroscience, University of Florida Brain Institute and Cancer Center, Gainesville, FL

Carlos D. Medina, BS

Neuro-Oncology Program and the Departments of Pediatrics, University of Florida Brain Institute and Cancer Center, Gainesville, FL

Karin B.N. Hoang, BA

Neuro-Oncology Program and the Departments of Pediatrics, University of Florida Brain Institute and Cancer Center, Gainesville, FL

M. Ian Phillips

nd Department of Physiology University of Florida Brain Institute and Cancer Center, Gainesville, FL

The overall goal of this review is to provide the pediatric neurologist with a theoretical foundation in gene therapy. Gene therapy became feasible in the early 1970s and the first transfer of a foreign gene into humans was approved by the NIH in 1989. Adenovirus, adeno-associated virus, herpes-simplex virus, retroviruses, and other vectors have been used to efficiently transduce genes into cells in vitro and in vivo. We discuss laboratory experiments that have provided a strong scientific rationale for implementing human clinical trials of gene therapy for neurologic malignancy. The development of viral and nonviral vectors that mediate efficient gene insertion into human cells has created the prospect of using gene therapy for cancer or brain disease. The NIH 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. (J Child Neurol 1997;12:1-12).

Journal of Child Neurology, Vol. 12, No. 1, 1-12 (1997)
DOI: 10.1177/088307389701200101


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