期刊
MOLECULAR GENETICS AND METABOLISM
卷 96, 期 4, 页码 151-157出版社
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/j.ymgme.2008.12.016
关键词
Gene therapy; Ornithine transcarbamylase deficiency; Clinical trial
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. (C) 2009 Elsevier Inc. All rights reserved.
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