Hemophilia Gene Therapy

First Gene Therapy Related Death Reported

Published in Gene Therapy Weekly, October 18th, 1999

An 18-year-old young man from Arizona died after undergoing experimental gene therapy to infuse a batch of genetically engineered viruses into his liver, according to a September 30, 1999, National Hemophilia Foundation report.

The boy had suffered from a serious genetic disorder, ornithine transcarbamylase (OTC) deficiency, that blocks the body's ability to break down ammonia and often results in death soon after birth. The experiment conducted on him had been controversial because his mild form of the disease had been well controlled by medicine and a strict diet.

The patient had reportedly volunteered to take part in the experiment in the...

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Source: Gene Therapy Weekly (1999-10-18)

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