Gene therapy: trials and tribulations - PubMed (original) (raw)

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Gene therapy: trials and tribulations

N Somia et al. Nat Rev Genet. 2000 Nov.

Abstract

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.

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