Gene therapy: trials and tribulations.
The art and science of gene therapy has received much attention of late. The tragic death of 18-year-old Jesse Gelsinger, a volunteer in a Phase I clinical trial, has overshadowed the successful treatment of three children suffering from a rare but fatal immunological disease. In the light of the success and tragedy, it is timely to consider the challenges faced by gene therapy--a novel form of molecular medicine that may be poised to have an important impact on human health in the new millennium.
Somia
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Laboratory of Genetics, Salk Institute, 10,010 North Torrey Pines Road, La Jolla, California 92037, USA.
Verma
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D000293:Adolescent; D000818:Animals; D002648:Child; D017321:Clinical Trials, Phase I as Topic / Q000592:standards; D000229:Dependovirus; D005260:Female; D015316:Genetic Therapy / Q000379:methods / Q000639:trends; D005822:Genetic Vectors; D006801:Humans; D012190:Retroviridae