Jac47,

 

Pardon my ignorance but how does one achieve systemic affects in genetics? 

 

In the case of Blood T-Cells I think I get it on the most superficial level.  It's ultimately a little like natural selection, and hopefully we will see that the Mutated T-Cells without the HIV-binding module will survive while their predecessor T-Cells ultimately die off.  Assuming that works,  mono-genetic diseases may be treatable systematically if they are found in blood stream cells.  I guess sickle-cell anemia comes to mind as another possibility. 

 

But lets say you are dealing with a genetic disease impacting the entire nervous system.  How would you achieve systemic affects?  Can you repair the DNA in one neuron and expect the change to carry-over to the rest of the body.  I have trouble with the concept.  Isn't this why the DN trials really only showed significant benefit to one or maybe 2 nerves.  Here we had some evidence of "Localized" Effects.

 

Sorry that you are doing the heavy lifting since Adenlyly left the board  (I hope he is successful with whatever venture he had on his mind, but hope he checks it too).  Anyways, thanks for helping us mere mortals understand the science and explaining the reasons for your enthusiasm.  I've lost a lot of money so far with SGMO, but I am really impressed but what I've seen to date and am more excited about the future of SGMO than ever, but certainly not content with they things are now.   I remain patient and cautiously optimistic that SGMO will prove its greatness but it will not be easy if the big corps feel threatened and want to snuff SGMO out.

 

Thanks again,

-BEHOLD