On September 17th, 1999, eighteen year old Jesse Gelsinger died as the result of his voluntary participation in a gene therapy experiment, becoming the first known human victim of this biotechnology [1]. Jesse suffered from ornithine transcarbamylase (OTC) deficiency, a rare metabolic disorder that prevents the correct metabolism of ammonia. This metabolic disorder affects 1 in 40,000 newborns [3]. Half of children with OTC die in their first month of life, and half of the survivors die before their fifth birthday. Jesse had a mild form of OTC and was therefore able to control the disease with diet and drugs.
The experimental protocol for which Jesse volunteered for through the University of Pennsylvania had no chance of providing him or any
…show more content…
It uses a combination of several converging bio technological approaches, moving it beyond traditional transplantation and replacement therapies [4]. Gene therapy is an experimental technique that uses genes to treat and prevent disease [10]. The research for gene therapy is thus driven by the hope of curing diseases currently incurable by modern medicine. From cancer to hemophilia, gene therapy is one of the most active and heavily pursued biomedical research fronts.
Gene therapy was ultimately the embryonic stem cell research of the 1990s; its ability to cure was thought to be boundless and the hype surrounding it unwavering, with promises in both therapeutic and financial realms. Billions of dollars stood to be made from curing diseases as rare as OTC and as common as cancer, leading to millions of dollars in investments [5]. It is thus important to consider the legal, social, and ethical implications of gene therapy and the profusion of genetic information that it will provide as this technique continues to emerge in the clinical and therapeutic