Lessons learned from the gene therapy trial for ornithine transcarbamylase deficiency

Mol Genet Metab. 2009 Apr;96(4):151-7. doi: 10.1016/j.ymgme.2008.12.016. Epub 2009 Feb 10.


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.

MeSH terms

  • Clinical Trials as Topic*
  • Clinical Trials, Phase I as Topic
  • Genetic Therapy*
  • Humans
  • Ornithine Carbamoyltransferase Deficiency Disease / genetics*
  • Ornithine Carbamoyltransferase Deficiency Disease / therapy*