gene therapy and murine leukemia retrovirus

Summer

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I'm not a scientist so please excuse if my question sounds ignorant.

I understand that murine leukemia retrovirus virus is being studied as possible vectors for gene therapy. Does anyone know if thhttp://www.facebook.com/pages/Whittemore-Peterson-Institute/154801179671?v=feed&story_fbid=168291079671is has actaully been tried in a human being please? thank you


http://books.google.com/books?id=8j...resnum=6&ved=0CCwQ6AEwBQ#v=onepage&q=&f=false

I do not know the answer to that question, but here is another article about murine leukemia retrovirus being used as a vectors. Look under "Types of viral vectors" then "Retroviruses".
 
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Thank you summer, I'll read it slowly to try to understand it - we could all do with degrees in molecular biology.
 
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Yes they've used it to cure bubble babies (SCID) and restore a working immune system both in Italy and here. New England Journal of medicine; Aiuiti in Italy and Candotti here. You need a virus as vector to bring the healthy gene into the genome. They took out some harmful parts and tested in animals for years but left in what are known as promoter sequences. Unfortunately when the virus carrying the healthy gene randomly integrated near a human oncogene in some cells it switched the oncogene on and tho the bubble babies were cured of SCID they eventually got cancer and had to be treated for that. Now they have removed those promoter sequences.

But this is unrelated to XMRV except perhaps it also switches on oncogenes thus perhaps accounting for higher risk of cancer but I have no idea
 

garcia

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Lorenzo's Oil disease cured by retrovirus

Someone asked about the use of retroviruses to cure disease recently. I came across this fascinating article in the Times today:

Gene therapy could cure Lorenzos Oil brain disease

A devastating brain disease that kills boys by the time of adolescence has been stopped in its tracks by a gene therapy that raises hope for thousands of families.

The treatment has halted the development of adrenoleukodystrophy (ALD) the genetic disorder featured in the film Lorenzos Oil in two Spanish boys aged 7 who would otherwise have suffered progressive and ultimately fatal brain damage.

The study is the first to show that a virus known as a lentivirus a disabled form of HIV can be used to correct genetic defects in human cells. This should assist the development of gene therapies for other diseases.

I never gave up hope of solving the riddle of my son Lorenzos ALD
by Augusto Odone.

Augusto Odone is a remarkable man:
"Neither of us was a scientist but we shared the conviction that every medical condition, when studied intelligently and with determination, could be resolved. In the face of much scepticism and even hostility, we decided to study ALD ourselves."

"Our culture prizes health and quality of life; too often this leads us to write off anyone who enjoys neither. This is not only selfish, it is short sighted. Science proves again and again that the impossible is just around the corner."
 
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The retrovirus did not cure the problem. Retroviruses are used as vectors to carry good genes into the stem cells and integrate into the genome there. They disable the infective parts of the virus. MLV (murine leukemia virus--similar to XMRV) has been used--but they prefer HIV for the reasons stated in the article.
 
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I'm not pedantic. They have to use viruses as vectors because viruses get into the cell, and retroviruses integrate into the genome. They used adeno-associated virus--which doesn't integrate into the genome--to treat a congenital disease that causes progressive loss of vision and blindness. Because cells in the retina stay for life, they didn't need to use a retrovirus.

The point of gene therapy is getting the correct gene into the cell. They are now working on other means than viruses but viruses will probably be the choice for many years to come.

What is the point here? It's an entirely different field and a very wonderful one that is finally making good progress but what does it have to do with CFS/lyme/ME--unless you want to speculate on the fact that we might have single gene disorders that could have rendered us vulnerable to lyme/CFS that could be corrected by gene therapy. But we probably don't. We have a constellation of relatively common polymorphisms most likely (gene variants) that have rendered us vulnerable to certain pathogens and toxins.