Jesse Gelsinger

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Jesse Gelsinger

Jesse Gelsinger (June 18, 1981 – September 17, 1999) was the first person publicly identified as having died in a clinical trial for gene therapy. Gelsinger suffered from ornithine transcarbamylase deficiency, an X-linked genetic disease of the liver, the symptoms of which include an inability to metabolize ammonia – a byproduct of protein breakdown. The disease is usually fatal at birth, but Gelsinger had a milder form of the disease, in which the ornithine transcarbamylase gene is mutated in only part of the patient's cells, a condition known as somatic mosaicism. As his deficiency was partial, Gelsinger managed to survive on a restricted diet and special medications.

Gelsinger joined a clinical trial run by the University of Pennsylvania that aimed at developing a treatment for infants born with the severe form of the disease. On September 13, 1999, Gelsinger was injected with an adenoviral vector carrying a corrected gene to test the safety of the procedure. He died four days later at the age of 18, on September 17, apparently having suffered a massive immune response triggered by the use of the viral vector to transport the gene into his cells, leading to multiple organ failure and brain death.[1]

A Food and Drug Administration (FDA) investigation concluded that the scientists involved in the trial, including the co-investigator James Wilson (Director of the Institute for Human Gene Therapy), broke several rules of conduct:

  • Inclusion of Gelsinger as a substitute for another volunteer who dropped out, despite Gelsinger's having high ammonia levels that should have led to his exclusion from the trial.
  • Failure by the university to report that two patients had experienced serious side effects from the gene therapy.
  • Failure to disclose, in the informed-consent documentation, the deaths of monkeys given a similar treatment.

The University of Pennsylvania later issued a rebuttal,[2] but the university and Children's National Medical Center each agreed to pay more than $500,000 to the government.[3] Both Wilson and the University are reported to have had financial stakes in the research.[4][5] After his death, all gene therapy trials in the United States halted for a time.[6] The Gelsinger case was a severe setback for scientists working in the field and a reminder of the risks involved.[7]


  1. ^ Stolberg, Sheryl Gay (November 28, 1999). "The Biotech Death of Jesse Gelsinger". The New York Times Magazine. Retrieved July 5, 2018.
  2. ^ "Institute for Human Gene Therapy Responds to FDA – Almanac Between Issues". 2000-02-14. Retrieved 2010-11-16.
  3. ^ Branca, MA (May 2005). "Gene therapy: cursed or inching towards credibility?". Nature Biotechnology. 23 (5): 519–21. doi:10.1038/nbt0505-519. PMID 15877060. S2CID 6628811.
  4. ^ Greenberg, Daniel S. Science for Sale. The Perils, Rewards, and Delusions of Campus Capitalism. Chicago: U. Chicago Press, 2007, 324pp., pages 104–106.
  5. ^ "Don't Compromise Ethics in Human Experiments, Bioethics Expert Says". 2008-04-18. Archived from the original on 2009-10-14. Retrieved 2010-11-16.
  6. ^ "Challenges in Gene Therapy". University of Utah. Retrieved 2020-11-02.
  7. ^ Wilson, James M. (2009-05-08). "A History Lesson for Stem Cells". Science. 324 (5928). 727–728. Bibcode:2009Sci...324..727W. doi:10.1126/science.1174935. PMID 19423804. S2CID 206520573. Retrieved 2012-02-29.

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