Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity


This was an excellent read, thank you for posting it.

Especially the part about under/overexpressed genes. It does share a lot of similarities to the Baylor study. But they concluded we don’t have a Cag-repeat disease IIRC.

I think we will need some scientist to go through a PFS patients body and brain post mortem if we want any define answers sadly.

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They had two in a brain bank in Texas 6 years ago they study.never heard anymore about it…

Amazing read but way above my understanding

Is this true? Why weren’t they studied?

That’s what I was told…but I’ve heard so much here over the past 6 years hard to keep track plus my brain is numb from this shit that doesn’t help…

What I’ve read of this paper seems quite amazing to me…Actually trying this therapy on rats but not humans…

It would seem similar dis order in pfs patients but working through an unknown mechanisms to cause this gene dysregulation not cag…

There seems to be some brains avaliable. But you need someone capable and willing to study it to.

I’d donate my brain, but I don’t live in the US and I probably won’t die for many years to come.

I guess we need to contact Dr. Peter Thompson surely after 6 years something was gained??

I’d guinea pig that ar45 also before I died…

Dr Thompson is just in charge of the brain bank right?

It’s up to scientists interested in PFS to contact them if I understand it right.

Might be worth asking if he sent any samples out though.

Nobody will then because the only people who seem interested in it are the ones who are dieing from it and their families

Good read from axo’s paper with relevance to this:

Crucially, Nedeslky et al. reported that wild-type AR of a 12Q polyglutamine-length, when expressed at very high levels, resulted in an degenerative phenotype indistinguishable from that caused by expansion of the AR polyglutamine tract

Where did you find this? I’d like to the full text, as “expressed at very high levels” is not very quantitative.