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Gene therapy gone wrong

The risks of gene therapy were realized in the 1999 case of Jesse Gelsinger , an 18-year-old patient who received gene therapy as part of a clinical trial at the University of Pennsylvania. Jesse received gene therapy for a condition called ornithine transcarbamylase (OTC) deficiency , which leads to ammonia accumulation in the blood due to deficient ammonia processing. Four days after the treatment, Jesse died after a massive immune response to the adenovirus vector. Barbara Sibbald. “Death but One Unintended Consequence of Gene-Therapy Trial.” Canadian Medical Association Journal 164 no. 11 (2001): 1612–1612.

Until that point, researchers had not really considered an immune response to the vector to be a legitimate risk, but on investigation, it appears that the researchers had some evidence suggesting that this was a possible outcome. Prior to Jesse’s treatment, several other human patients had suffered side effects of the treatment, and three monkeys used in a trial had died as a result of inflammation and clotting disorders. Despite this information, it appears that neither Jesse nor his family were made aware of these outcomes when they consented to the therapy. Jesse’s death was the first patient death due to a gene therapy treatment and resulted in the immediate halting of the clinical trial in which he was involved, the subsequent halting of all other gene therapy trials at the University of Pennsylvania, and the investigation of all other gene therapy trials in the United States. As a result, the regulation and oversight of gene therapy overall was reexamined, resulting in new regulatory protocols that are still in place today.

  • Explain how gene therapy works in theory.
  • Identify some risks of gene therapy.

Oversight of gene therapy

Presently, there is significant oversight of gene therapy clinical trials. At the federal level, three agencies regulate gene therapy in parallel: the Food and Drug Administration (FDA), the Office of Human Research Protection (OHRP), and the Recombinant DNA Advisory Committee (RAC) at the National Institutes of Health (NIH). Along with several local agencies, these federal agencies interact with the institutional review board to ensure that protocols are in place to protect patient safety during clinical trials. Compliance with these protocols is enforced mostly on the local level in cooperation with the federal agencies. Gene therapies are currently under the most extensive federal and local review compared to other types of therapies, which are more typically only under the review of the FDA. Some researchers believe that these extensive regulations actually inhibit progress in gene therapy research. In 2013, the Institute of Medicine (now the National Academy of Medicine ) called upon the NIH to relax its review of gene therapy trials in most cases. Kerry Grens. “Report: Ease Gene Therapy Reviews.” The Scientist , December 9, 2013. http://www.the-scientist.com/?articles.view/articleNo/38577/title/Report--Ease-Gene-Therapy-Reviews/. Accessed May 27, 2016. However, ensuring patient safety continues to be of utmost concern.

Questions & Answers

what z microbiology
Mwelwa Reply
microbiology is all about the scientific study of microorganisms, their life changes, their life span, structural punctuality and their phenomenon.
Ridwan
thanks
micah
Best scope in microbiology
Rahul
wat abut skin
Elyas
what happened when someone died and what happened to the blood
Franca Reply
nice question
Attari
when you die you have no memory and the blood dry off
Falere
what microorganisms is all about?
Ridwan
And how many does the microorganisms is classified?
Ridwan
what is endoparasites and exo parasite and their example
OGEDE Reply
Endo : inside the body Exo/ecto : outside or on the body
Paul
examples please
OGEDE
Louse for exo/ecto Helminths (worms) for endo
Paul
what's prokaryotic
mohaiminul Reply
A prokaryotic cell is a cell with no membrane bounds organelles
Paul
what are the main effect of parasite?
yunusa Reply
nutritional group of micro organism
Mana Reply
what are the charateristic bacteria, eukaryotes, and viruses have in commom
Ami Reply
D N A ar purno rup ki
Ruba Reply
বাংলায় দেখবো DNA এর পূর্রন্যরুপ কি
Ruba
what's microbiology
micah
micro biology is the study of micro organisms,those being unicellular multicellular or a cellular
aniisha
deoxyribonucleic acid is a long molecule that contains our unique genetic code.
aniisha
Does the parasite/parasitology are under microbiology or not
yunusa
Define bacteria
Kainat
Bacteria are a type of biological cell & they constitute a large domain of prokaryotic microorganisms.
raisa
what kind of microbiology
Mira
hello what is main function of T and B cells
Ishaq Reply
T cells are involved in cell-mediated immunity while B cells are primarily responsible for humoral immunity (relating to antibodies)
Janet
what is hepatitis B
Sunday
it's viral infection that attacks the liver and can cause both acute and chronic diseases. the virus is most commonly transmitted from mother to child during birth and delivery, as well as through contact with blood or other body fluids...
Dejene
What are fundamental experiment of microbiology?
Lucky Reply
study of microscopic organisms, unicellular and multicellular
Falere
what are the characteristics of bacteria, viruses, and eukaryotes
Ami
bacteria-are living organism. contain a peptidoglycan/lipopolysaccharide cell wall. VIRUS-are considered as organic structure which interact with living organism don't have acell wall. they have many differents like bacteria size -large(1000nm) virus size-smaller(20-400nm). bacteria ribosomes presen
Dejene
virus ribosomes absence. bacteria living organism. virus b/n living and non-living things
Dejene
eukaryotic cell has a true membrane bond nucleus and has other membranous organelles that allow for compartmenalization of function
Dejene
This answer is wrong
Jeffrey Reply
who discovered the growth curve
Joy Reply
Example of coccus bacteria
Nweke Reply
staphylococcus aureus
Elton
MRSA any bew information
Satish
pl. read any new information about MRSA
Satish
Any stru tural finding about novel corona virus
Satish
Examples of spiral bacteria
Nweke Reply
Example of Bacillus bacteria
Nweke
pusals
manikanta
another one
Nweke
what is the desifection decontamination or sterilization
Sahra Reply
Is the removal of dangerous substances or germs from an area, objects or person
Florence
It is the removal of any pathogen, including their spores
Paul
removal of all forms of life including endospore
abdulbasid
I think there is a difference between disinfection and sterilization... we should consider those....
Mosimanegape
disinfection deals with removing harmful microbes at the surface level it doesn't destroy or kill spores ( it uses oxidizing agents ,alcohols , phenolic comp ,aldehydes etc) but sterilization deals with total elimination of all forms of lives.(this uses heat, steam, radiation and chemicals)
Onyinye
disinfecting is preventing microorganisms while sterilization is killing of microorganisms completely
Janet

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Source:  OpenStax, Microbiology. OpenStax CNX. Nov 01, 2016 Download for free at http://cnx.org/content/col12087/1.4
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