eported to the NIH. But we will never know whether or not these patients died as a direct effect of their treatment.The most recent, and most publicized death that occurred because of gene therapy was the death of Jessie Gelsinger. Jessie appeared from the outsider to be a healthy 18-year-old boy. His only brush with death came in 1998 when he fell into a coma. However, Jessie was not entirely healthy. He was on a low protein diet and took a pile of pills every day. He had Ornithine Transcarbamylase Deficiency. This is a genetic disorder caused by a deficiency of one of the enzymes in the urea cycle. The urea cycle involves a series of steps that takes place in the liver, in which nitrogen is removed from the blood and converted into urea. Each step requires a specific enzyme and when one is missing nitrogen builds up and is converted into ammonia, which is highly toxic, instead of urea. Ammonia then can reach the brain through the blood, where it may cause brain damage and even death. Jessie did not die from this disorder though. He died from the treatment he voluntarily received from the University of Pennsylvania.The treatment began in September of 1999. It included directly inserting an adenovirus into the hepatic artery that leads directly into the liver. This specific treatment was much looked down upon by the NIH because it was dangerous. But, after many public discussions between the University of Pennsylvania researchers and the NIH, the NIH eventually voted for approval of the study. Jessie died only four days after the start of his treatment. His organs failed one at a time, starting with jaundice and then blood clots, kidney failure, lung failure and lastly, brain death. Since Jessie was the supposedly the first patient to have ever died as a direct result of gene therapy, his death sent out a lot of alarming messages about this treatment that was new to much of the public. The news reporters went wild in ...
