Published in Gene Therapy Weekly, October 18th, 1999
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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