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From Wikipedia, the free encyclopedia

Jesse Gelsinger (June 18, 1981 - September 17, 1999) was the first person publicly identified as having died in a clinical trial for gene therapy. Gelsinger suffered from ornithine transcarbamylase deficiency, an X-linked genetic disease of the liver, the symptoms of which include an inability to metabolize ammonia - a byproduct of protein breakdown. The disease is usually fatal at birth, but Gelsinger had a milder form of the disease, in which the ornithine transcarbamylase gene is mutated in only part of the patient's cells, a condition known as somatic mosaicism. As his deficiency was partial, Gelsinger managed to survive on a restricted diet and special medications.

Gelsinger joined a clinical trial run by the University of Pennsylvania that aimed at developing a treatment for infants born with the severe form of the disease. On September 13, 1999, Gelsinger was injected with an adenoviral vector carrying a corrected gene to test the safety of the procedure. He died four days later at the age of 18, on September 17, apparently having suffered a massive immune response triggered by the use of the viral vector to transport the gene into his cells, leading to multiple organ failure and brain death.

A Food and Drug Administration (FDA) investigation concluded that the scientists involved in the trial, including the co-investigator James Wilson (Director of the Institute for Human Gene Therapy), broke several rules of conduct:

Inclusion of Gelsinger as a substitute for another volunteer who dropped out, despite Gelsinger's having high ammonia levels that should have led to his exclusion from the trial.

Failure by the university to report that two patients had experienced serious side effects from the gene therapy.

Failure to disclose, in the informed-consent documentation, the deaths of monkeys given a similar treatment.

The University of Pennsylvania later issued a rebuttal, but the university and Children's National Medical Center each agreed to pay more than $500,000 to the government. Both Wilson and the University are reported to have had financial stakes in the research. After his death, all gene therapy trials in the United States halted for a time. The Gelsinger case was a severe setback for scientists working in the field and a reminder of the risks involved.

1. ^ Stolberg, Sheryl Gay (November 28, 1999). "The Biotech Death of Jesse Gelsinger". The New York Times Magazine.

2. ^ "Institute for Human Gene Therapy Responds to FDA - Almanac Between Issues". Upenn.edu. 2000-02-14.

3. ^ Branca, MA (May 2005). "Gene therapy: cursed or inching towards credibility?". Nature Biotechnology. 23 (5): 519-21. doi:10.1038/nbt0505-519. PMID 15877060. S2CID 6628811.

4. Greenberg, Daniel S. ^ Science for Sale. The Perils, Rewards, and Delusions of Campus Capitalism. Chicago: U. Chicago Press, 2007, 324pp., pages 104-106.

5. ^ "Don't Compromise Ethics in Human Experiments, Bioethics Expert Says". Law.virginia.edu. 2008-04-18. Archived from the original on 2009-10-14.

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From Wikipedia, the free encyclopedia Jesse Gelsinger (June 18, 1981 - September 17, 1999) was the first person publicly identified as having died in a clinical trial for gene therapy. Gelsinger suffered from ornithine transcarbamylase deficiency, an X-linked genetic disease of the liver, the symptoms of which include an inability to metabolize ammonia - a byproduct of protein breakdown.

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1. 4 year old girl became the first gene therapy patient on September 14, 1990. She had adenosine deaminase deficiency, a genetic disease which leaves her defenseless against infections
2. Gene Therapy is short-lived, meaning multiple treatments are needed for a desired affect as of now.
3. 1999- Jesse Gelsinger became the first person to die whilst undergoing a gene therapy treatment
4. Gendicine is the world's first commercial gene therapy that uses a virus to insert the gene into the human genome
5. For success in Gene Therapy, the follwing must occur:
1) the gene must be known
2) the gene must be able to be expressed
3) the gene must be introduced into the appropriate host tissue

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Gene therapy is the process of replacing defective genes in a cell with healthy ones. In simple terms, genes form the basis of heredity. They come as triplets of nitrogenous basis which form amino acids. Amino acids are a composition of proteins which play a crucial role in the functioning of our bodies. Therefore defective genes cause malfunctions in metabolic pathways which become diseases, of which the causes are deeply rooted in our genetic design. Currently there isn't a cure for genetic disorders and treatment is only indicative. This is why there's so much turmoil in the medical arena about the possible effects of gene therapy.

Gene Therapy Treatment

Gene therapy can be categorized in to two different types-somatic cell therapy and reproductive cell also referred to as germline therapy. Somatic cells involves targeting somatic cells for gene replacement, while reproductive cell therapy involves replacing defective genes in reproductive cells with correct genes. Changes made in the genetic makeup of somatic cells is only corrective for the patient. Alterations are however not inherited by offspring of the treated person.

List of Pros of Gene Therapy

1. Untapped Potential
One notable factor that gives gene therapy the edge is the remarkable therapeutic potential it has.

2. Replace Defective Cells
Humans have always been prone to variety of diseases; as one cure is discovered, another disease emerges. We are constantly attacked by newer, more dangerous and vituperative types of germs and pathogens. Although many of such diseases can be treated or cured medically, there is no cure for genetic disorders unless defective cells are replaced by proper ones which is what gene therapy does.

3. Eradicate Disease
Furthermore, targeting reproductive cells can get rid of such defects for good. People with genetic disorders such as Alzheimer's disease, Huntington's disease and Parkinson's disease are among those who require gene therapy.

List of Cons of Gene Therapy

1. Modify Human Capabilities
One con of gene therapy is that it may be used to enhance and modify human capabilities. If this was feasible, standards for normal human life would be altered for good. Even if certain countries experimented with this form of therapy, they could create unstoppable armies much like in those movies we've seen on the big screen.

2. Damage Gene Pool
If gene therapy was performed to a certain degree, it possibly could permanently change the human gene pool. Another thing would also be-this treatment therapy may possibly be for the rich only, and could make the rich more richer and the poor more poorer, indefinitely.

3. Rise in Disorders
While the most common choice as a vector, using the virus for such a purpose is not guaranteed. There's an exact point in the host genome where the right should be brought in and there are no assurance that the viral enzyme responsible for this step will be able to bring in the right genes at the exact point in the host genome. If there's an error in the process, the results could be bring about severe disorders. In addition, the body may destroy the vector perceiving that it is a foreign body.

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Gene therapy is the process of replacing defective genes in a cell with healthy ones. In simple terms, genes form the basis of heredity. They come as triplets of nitrogenous basis which form amino acids. Amino acids are a composition of proteins which play a crucial role in the functioning of our bodies.

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Explore the what's and why's of gene therapy research, includingan in-depth look at the genetic disorder cystic fibrosis and how gene therapy could potentially be used to treat it.

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The vector can be injected or given intravenously (by IV) directly into a specific tissue in the body, where it is taken up by individual cells. Alternately, a sample of the patient's cells can be removed and exposed to the vector in a laboratory setting.

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California Institute for Regenerative Medicine

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Gene Therapy and Cystic Fibrosis Animation

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Gene Therapy and Cystic Fibrosis Animation

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This clever little animation explains the process of gene therapy for Cystic Fibrosis ... and it was designed by Changing Futures participant Caitlin. This film is part of the Changing Futures Project: www.changing-futures.co.uk.