dear paula,
so what happened to the great stem cell promise? gone with the wind.
because, it must be illegal to have a cure for people.
if you think that's alot of cash for rats, then ask
Greg G. at the U of Kentucky -how much old lady rhesus monkeys go for -

in the $100,000 category -? maybe higher now...
luv
tena
nothing new under the sun...

The Dangerous Promise of Gene Therapy
By Sophia M. Kolehmainen
Gene therapy has not yet proven successful in curing disease.
In the mid-1980's scientists began to extol the promises of gene-therapy. Conceptually (and if you consider the world only at the sub-microscopic level), gene therapy is a logical, straightforward solution to genetic disease: if a gene seems to be causing a disease, then to cure the disease scientists must remove the "bad" gene, and substitute or add a "good" gene. The reality is much more complex. Though more than three hundred gene therapy protocols, involving more than four thousand patients, have been approved for human trials in the United States, gene therapy has yet to fulfill its promise of curing any genetic disease.
Jesse died because of a gene therapy experiment.




Jesse: gene therapy gone wrong

On September 17th, 1999, eighteen-year-old Jesse Gelsinger died as a result of his voluntary participation in a gene-therapy experiment, becoming the first known human victim of this technology. Jesse's experience illuminates important elements in gene therapy that should make government agencies, scientists, and the public take the need to regulate and oversee this technology very seriously.

Jesse had a rare genetic disease, known as ornithine transcarbamylase (OTC) deficiency, which affected his ability to rid his body of ammonia, a usual, but toxic, breakdown product of protein. Half of children with OTC die in their first month of life, and half die before their fifth birthday. Jesse had a mild form of the disease because some of his enzymes were functioning normally. He was therefore able to control the disease with diet and drugs, though he needed to take 32 pills a day.
Ironically, Jesse would not have benefited from the experiment.



The therapy was intended to help babies with a rare genetic disease.







The experimental protocol for which Jesse volunteered had no chance of providing him, or any of the other volunteers, with any benefit.
It was designed only to test the safety of a treatment that would be used on babies with the fatal form of OTC.
The scientists who designed the protocol at the University of Pennsylvania's Institute for Gene Therapy, Dr. James Wilson and Dr. Mark Batshaw, believed that OTC could be surmounted with gene therapy.
They hoped to infuse babies who had OTC with genes that would help them produce the missing enzymes.
In order to get these genes into the patient's cells, Dr. Wilson developed a weakened cold virus (known as adenovirus) which was designed to enter the cells as any virus would, but, instead of delivering disease, it was supposed to deliver the corrective OTC gene.
Wilson and Batshaw hoped that the infusion of adenovirus and corrective genes could be used to reduce infant fatalities by controlling the high levels of ammonia in babies with OTC immediately after birth.




Doctors did not foresee serious side effects or fatality.
Wilson and Batshaw worked together to develop the OTC protocol and, in 1995, they submitted it to the National Institute of Health's (NIH) Recombinant DNA Advisory Committee (RAC) and the Food and Drug Administration (FDA) for review and approval, as is required for all human experiments involving gene therapy.

Jesse was deemed eligible for the study and assigned to the final test subject group--the group that would receive the highest dose of adenovirus. At the time, the researchers believed that in the worst case, the trial might result in an inflamed liver. On September 13, 1999 Dr. Raper injected 30 milliliters of the adenovirus with the corrective OTC gene into Jesse's bloodstream. According to the physicians, Jesse's severe immune system reaction led to multiple-organ-system failure and he died on September 17th, 1999, four days after the gene-therapy injection.

Some test animals died before human trials.



Some reviewers were concerned but the FDA approved the trials.

The safety of gene therapy

Human gene therapy trials raise the questions:
http://www.actionbioscience.org/biot...lehmainen.html