Gene therapy: Trials and tribulations

Nikunj Somia, Inder M. Verma

Research output: Contribution to journalReview articlepeer-review

562 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)91-99
Number of pages9
JournalNature Reviews Genetics
Issue number2
StatePublished - Nov 2000


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