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in vivo gene therapy..help!

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in vivo gene therapy..help!

Postby christie » Mon May 16, 2005 8:20 pm

is in vivo gene therapy permanent or not??(specifically concerning cystic fibrosis!)why is it or why isn't it?? help! :(
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Postby mith » Mon May 16, 2005 8:35 pm

According to this site, it does not work at the present moment
http://www.cfgenetherapy.org.uk/genetherapy.htm

If it does work though, large number of cells will need to be infected with the virii or liposomes. If enough are infected, the effect should be permanent.
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Postby christie » Tue May 17, 2005 8:26 pm

the info on the site doesn't quite satisfy me...thnx anyway. if you have an answer to my question please answer me A.S.A.P. !!thnx!
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Postby Chris4 » Tue May 17, 2005 9:57 pm

I think I understand what you are asking now. If you had gene therapy would it be permanent as would you survive the illness forever as in the new gene be in your cells forever.
Germline gene therapy is permanent in an organism but that hasnt been done yet in humans as its highly controversial.
I think ex vivo also allows permanent integration of the gene into the target cells. according to this: http://www.esgt.org/downloads/position_2.pdf

I think your answer is it can be either temporary or permanent. Not the answer you were looking for, but thats often the way in science :lol:
http://www.deanlab.org/research3.html - This site says can be permanent or transient depending on vector design.
When the cell divides sometimes the DNA may not be maintained and sometimes it is.

Retroviruses are the only vectors which appear to give a permanent change. http://www.gvt-journal.com/content/2/1/9

My summary - Its all depends on the vector design and according to the last article (which I think is a very good source), Retroviruses are what you need to use for pernament changes.

I hope thats what your looking for. I dont particulary like genetics (its too hard), but i just learnt a lot about gene therapy. :D

P.S. Just read your post again. Cystic Fibrosis. :arrow: According to this patent application there are adenoviruses for CF.
http://www.pharmcast.com/Patents100/Yr2 ... 040505.htm
But they therefore cant be permenent.

The adenovirus is used because it naturally affects the airways. http://www.ndsu.nodak.edu/instruct/mccl ... %20Vectors
According to that tho, you only need 5 -10% of the cells to take up the gene as they connect electrically.

http://www.ingentaconnect.com/content/a ... 5/art00005
Another source for CF.
So adenoviruses can be permanent if you get the gene into enough of the cells.

I better stop there or it will get too complicated and i'll never get to sleep. :roll:
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