Gene Therapy Clinical Trials in the Human Brain
The longstanding promise of gene therapy of all types has yet to be fully realized. After a difficult period, however, developments in recent years have created a resurgence of interest in the clinical utility of this unique technology. To the casual observer, the number of studies in the brain which have entered clinical trials is often surprising. The excellent safety record of the numerous studies outlined above and several others not reviewed here, combined with encouraging preliminary efficacy data suggests that gene therapy will continue to evolve as a therapeutic option in clinical trials for CNS disease. With the explosion in human trials for these disorders, it is very possible that the next few years will finally see sufficient success in advanced trials of one or more approaches to justify FDA approval and use in general clinical practice.
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