Gene therapy: trials and tribulations
Top Cited Papers
- 1 November 2000
- journal article
- review article
- Published by Springer Nature in Nature Reviews Genetics
- Vol. 1 (2) , 91-99
- https://doi.org/10.1038/35038533
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.Keywords
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