“We're still working on proof of concept in animals,” Dr. Mahley told Neurology Today. “We have to establish safety, and it will be 18-24 months before we will have a structure corrector that we can use in humans.”Human tests would begin with people who have suffered traumatic brain injuries, such as soldiers and athletes, he said. If they have better outcomes than those not treated, that would be considered evidence of PY-101 blunting the toxic effects of APOE4.
PY101 Update
PY101 Update
http://journals.lww.com/neurotodayonlin ... UAL.9.aspx
Re: PY101 Update
Thanks for sharing, ApropoE4. This holds much promise for us. Fingers crossed...and hurry 
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Gilgamesh
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Re: PY101 Update
Yes, this is great news. The work with small-mol. correctors is the most promising out there, I believe. (Just look at my avatar image!! 
)
The question may boil down to delivering it in such a way that it reaches all, or most parts of the brain in sufficient quantities. Having the right pharmacokinetics will also help, though I wouldn't mind having to take a pill every hour, if it proved necessary (because of rapid clearance, for ex.).
This is exciting! Deals with the root of the problem. Well, almost. The next step -- which researchers are also working on -- is changing the gene itself. That's the root of the problem! With that solved, we can close up shop here at apoe4.info.
Onward!
GB
)The question may boil down to delivering it in such a way that it reaches all, or most parts of the brain in sufficient quantities. Having the right pharmacokinetics will also help, though I wouldn't mind having to take a pill every hour, if it proved necessary (because of rapid clearance, for ex.).
This is exciting! Deals with the root of the problem. Well, almost. The next step -- which researchers are also working on -- is changing the gene itself. That's the root of the problem! With that solved, we can close up shop here at apoe4.info.
Onward!
GB
Re: PY101 Update
G.
This is great news but your hope for gene therapy is not enough to rid AD from the human race. There are other genes, maybe up to 20, last count, that influence getting AD and there are many without the dreaded ApoE4 that get AD.
Bentkat
This is great news but your hope for gene therapy is not enough to rid AD from the human race. There are other genes, maybe up to 20, last count, that influence getting AD and there are many without the dreaded ApoE4 that get AD.
Bentkat
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Gilgamesh
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Re: PY101 Update
Hey Bentkat. You're absolutely right about Alzheimer's! But by "the problem" I meant the problem of ApoE4-associated pathology.
But when effective gene therapy arrives, it will likely be generalizable to all genes, or so I hope! I've got a long list of genes I'd like to correct.
By the way, great, unusually clear explanation of why ApoE4 is so not fun:
http://labs.gladstone.ucsf.edu/huang/pa ... e-research
GB
But when effective gene therapy arrives, it will likely be generalizable to all genes, or so I hope! I've got a long list of genes I'd like to correct.
By the way, great, unusually clear explanation of why ApoE4 is so not fun:
http://labs.gladstone.ucsf.edu/huang/pa ... e-research
GB
