Gene therapy: trials and tribulations

N Somia, IM Verma - Nature Reviews Genetics, 2000 - nature.com
N Somia, IM Verma
Nature Reviews Genetics, 2000nature.com
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.
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.
nature.com