Author Topic: Gene Therapy takes a step forward...  (Read 1443 times)

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Offline Flipside

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Gene Therapy takes a step forward...
http://www.bbc.co.uk/news/health-11313273

Good news on the medical side of things, looks like after a shaky start things are moving forward. Odd that one death can be considered a shaky start though if you consider how many people we killed getting medicine where it is today, but then, I suppose from the point of view of the family, that doesn't really count...

 

Offline MP-Ryan

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Re: Gene Therapy takes a step forward...
Gene therapy is risky - the death was due to cancer - and it's used on patients that can survive without it but have virtually no quality of life.  Jesse's death was a setback because the chances of cancer occurring were initially thought to be fairly low.  We've since learned otherwise.

The problem is that gene therapies in humans are unable to use targeted insertions (e.g. full gene replacement through recombination).  Instead, we're forced to use viral vectors, typically adenoviruses, that insert randomly.  Then we hope that it fully inserted the target gene and it's regulating sequences (if necessary) - the cells that test out positive are re-implanted in the patient.  Trouble is, that insertion may have disrupted another gene, turning it from protooncogene into full oncogene, from where it causes cancer.  All patients who received gene therapy right now - limited primarily to Severe Combined Immunodeficiency (SCIDs), thalassemias, and other severe blood/immune disorders because of the risks -  have a risk of developing cancer.

Once someone works out the vector/insertion problem to get reliable results with a targeted insertion, gene therapy will be a lot less risky because the chances of insertion in a protooncogene will be more or less eliminated.

Regardless, this is good news.  Thalassemias are nasty.
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Offline General Battuta

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Re: Gene Therapy takes a step forward...
Gene therapy is risky - the death was due to cancer

I was under the impression it was due to a massive immune reaction to the viral vector. I know cancer is a big risk, but didn't Jesse Gelsinger (spelling?) die of organ failure?

 

Offline MP-Ryan

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Re: Gene Therapy takes a step forward...
Gene therapy is risky - the death was due to cancer

I was under the impression it was due to a massive immune reaction to the viral vector. I know cancer is a big risk, but didn't Jesse Gelsinger (spelling?) die of organ failure?

Now I'm going to have to look it up.  I swear my prof in Human Genetics lectured about it and said cancer, but that WAS a few years ago and my memory has been faulty before =)

...and a quick search on PubMed indicates you are correct, sir.  I was thinking of the SCIDs gene therapy trials, while Jesse was for an X-linked gene mutation.

I bow to your superior memory  :yes:
"In the beginning, the Universe was created.  This made a lot of people very angry and has widely been regarded as a bad move."  [Douglas Adams]

  

Offline General Battuta

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Re: Gene Therapy takes a step forward...
The cancer risk is still out there, though. They even said this successful gene therapy could still cause leukemia.