Lessons learned from the gene therapy trial for ornithine transcarbamylase deficiency.
It has been 9 years since Mr. Jesse Gelsinger died from complications of vector administration in a liver gene therapy trial of research subjects with a deficiency of ornithine transcarbamylase (OTCD). This study was performed at the Institute for Human Gene Therapy of the University of Pennsylvania (Penn) which I directed. His tragic death provoked a series of events that had implications beyond those directly involved in the clinical trial. The events surrounding the death of this research subject have been the topic of much coverage and commentary in the popular press. The goal of this article is to share with you my reflections on the OTCD gene therapy trial and lessons that I have learned which may be of value to others engaged in various aspects of translational medicine.
Wilson
James M
JM
Department of Pathology and Laboratory Medicine, University of Pennsylvania, Suite 2000 TRL, 125 S. 31st Street, Philadelphia, PA 19104-3403, USA. wilsonjm@mail.med.upenn.edu
D002986:Clinical Trials as Topic; D017321:Clinical Trials, Phase I as Topic; D015316:Genetic Therapy; D006801:Humans; D020163:Ornithine Carbamoyltransferase Deficiency Disease / Q000235:genetics* / Q000628:therapy*