Gene Therapy Death Prompts Review of Adenovirus Vector

Abstract

For the past 3 months, faculty and staff members at the University of Pennsylvania9s Institute for Human Gene Therapy have been trying to understand why a relatively fit 18-year-old with an inherited enzyme deficiency died on 17 September, 4 days after doctors at Penn injected a genetically altered virus into his liver. Last week, they presented their data to a government advisory committee. The patient was the first in a gene therapy trial to die of the therapy itself; his death is the latest blow to a field that has been struggling to live up to the promise and hype surrounding the first gene therapy trials a decade ago.