Trying to wrap my head around the potential re-administration of AAV with chimeric AAV | SGMO Message Board Posts

Sangamo Therapeutics, Inc.

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Msg  128487 of 130734  at  6/19/2019 3:36:25 PM  by

GAGholder


 In response to msg 128485 by  gg100
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Re: Trying to wrap my head around the potential re-administration of AAV with chimeric AAV

Ill post the abstracts which got my brain moving. What SM said in terms of redosing was outside of my radar when I made this topic. From what Ive read, and the experiments have shown is that a vector, say, aav5 can be modified to express a polypeptide from a different aav, say aav1, and neither aav1NABS nor aav5NABS will do much about it. Further, transduction (in animals) seem to have similar results in the form of transfection as the unmodified version of the aav. When they redosed with those chimeric aavs they found that there were NABS developed to them and would need to use a different chimeric aav.

To me if redosing aavs is possible and expression is the same, the durability still wins the day. Im not sure this would allow you to redose indefinitely but to be able to do it once would be neat.


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