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Journal of Child Neurology
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Topical Review: Gene Therapy for Neurologic Disease: Benchtop Discoveries to Bedside Applications. 2. The Bedside

Bernard L. Maria, MD, MBA

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

Carlos D. Medina, BS

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

Karin B.N. Hoang, BA

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

M. Ian Phillips, DsC

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

Important advances in basic research have made it possible to examine the safety, toxicity, and efficacy of gene therapy in humans for over 5 years. The development of sophisticated gene delivery systems has resulted in approval by the Recombinant DNA Advisory Committee (RAC) of 125 gene therapy or gene marking studies. One of the primary applications of current retroviral-mediated gene insertion technology has been for malignant brain tumors. Studies are therefore underway to examine the efficacy of "suicide" gene therapy in children with recurrent brain tumors and adults with newly diagnosed or recurrent gliomas. Since a high proportion of genetic disorders produce neurologic dysfunction, gene therapy is likely to impact the management of neurologic disease in the foreseeable future. Patients with human immunodeficiency virus (HIV), Gaucher's disease, and Hunter syndrome are now enrolled in gene therapy trials. It will be challenging for the child neurologist to stay abreast of rapid developments in the field of gene therapy. By participating in the design and implementation of clinical trials in gene therapy, the neurologist may reduce the intense toll that several neurologic diseases take on children and their families. (J Child Neurol 1997;12:77-84).

Journal of Child Neurology, Vol. 12, No. 2, 77-84 (1997)
DOI: 10.1177/088307389701200202
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