Re: [DIYbio] Re: Curious about zink finger nuclesases / gene therapy

If the plasmid encodes a replacement and a CRISPR/TALEN it could certainly replace endogenous genes, but you'd have to watch put for copy number then as you could end up with plasmid encoded plus two chromosomal! Also, prolonged expression of DNA modifying enzymes would, I feel, be a cancer risk as they never have 100% specificity..

Ravasz <ravaszmeister@gmail.com> wrote:
For my PhD I've been designing plasmids which replicate like a chromosome and can be maintained extrachromosomally (even replicated and delivered to the daughter cells upon proliferation) in the human cell. This completely avoids any integration issues, but it has only been tested in cell cultures.

Of course this method does not allow replacing genes, only adding new ones, but still has a lot of potential IMO.

On Wednesday, 26 September 2012 09:54:39 UTC+2, Mega [Andreas Stuermer] wrote:
Hi,


I read about zink finger nucleases, and that they may be used in living organisms to alter their genome (after birth, as an adult) . 

I was wondering how that was posssible, how do you get the enzyme in every cell of the body? Is it just injected in the blood stream and then gets distributed automatically? 
Or do you inject it into the organ needed? But how can it penetrate the cell wall? Is it so small that it fits through the celll membranes? 




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