Jesse Gelsinger had a scary brush with death. The teenager had stopped taking his medications, was having bouts with nausea and vomiting and eventually found himself curled up listlessly on the couch, 15 pounds underweight and scared. http://www.genelet

ter.org/05-01 00/features/gelsing er1.html

By the time his parents got him to the hospital, his metabolic processes had careened out of control. He fell into a coma and a few days later, he stopped breathing in his father's arms.

Jesse suffered from a disorder called partial ornithine transcarbamylase (OTC) deficiency, which kept him perpetually in danger of hoarding toxic levels of ammonia in his blood. Half of all infants born with the condition die within a month.

As soon as he turned 18, Jesse Gelsinger volunteered for a gene therapy trial to for OTC and died as a result of the procedure.

In Jesse’s case, a replacement gene for OTC was packaged into an engineered adenovirus, which was injected in high dose, directly into his liver.

The gene carrier, or vector, was the prime suspect in Gelsinger's death, since the adenovirus can cause liver and lung damage. (The virus can trigger widespread blood clotting and inflame surrounding tissue.)

Why did they choose adenovirus? It can be grown easily in great amounts.

They have engineered versions with deleted genes E1 and E4.

It is good at infecting human cells.

In response to his death, the NIH asked researchers all over the country and found more than 650 dangerous adverse reactions they also had previously kept secret, including several deaths.

A Canadian Story ……… this is a case of “Suicide Gene Therapy” cbc.ca/national/ magazine/gene/

James Dent had a malignant brain tumor. With little hope of conventional treatment, Dent signed a consent form for gene therapy, which did present a risk of death. There was no mention of patients who suffered serious adverse events relevant to his experiment.

James Dent would be treated in two stages. The first consisted of tumor surgery and gene therapy. He was injected with an adenovirus vector, carrying the Herpes Simplex thymine kinase gene. This was injected directly into his brain tumor.

In the meantime, he returned home. ……Two weeks later, he began the second stage of the experiment, the injection of the antivirus drug ganciclovir… Within two days, he he went into septic shock and died.

Only days before James Dent began the second stage of his gene therapy treatment, a patient died in Indiana during the same experiment. That death was reported to U.S. regulators as unexpected and possibly related to the gene therapy.

Gene therapy has about a 10 year history. To date, over 4,000 people have participated in over 350 gene therapy clinical protocols (25% with adenovirus), with at best limited success.

By far, most of the clinical trials involve gene therapy for cancer. About 100 such trials are currently ongoing.

The major limitation of gene therapy in general, seems to be a limitation in being able to deliver enough of the “engineered gene.”