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Huntington's disease treated for first time

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Re: Huntington's disease treated for first time

#52
"If one of your parents has Huntington's disease, there's a 50% chance that you will inherit the altered gene and will eventually develop Huntington's too."

Have they never heard of genetic diagnostics? For example with a combination of preimplantation generic testing and in-vitro fertilization you can prevent passing on known genetic mutations to the next generation.

Re: Huntington's disease treated for first time

#53

Earlier quoted context omitted.

> Scientists need better messaging or else we're going to stop having breakthroughs like this Sure, but it's really sad that scientists need to justify their funding to the public - they already spend so much time justifying it to the NIH and others for funding. So many people have had their careers jeopardized by finding pulled mid-project. I am really concerned about our research pipeline, because my post-doc frien…

[flagged]

You missed the second part of that sentence buddy.

Your examples would match if every base commander had to write public messaging on their bases’ budgets and projects.

Re: Huntington's disease treated for first time

#54

What part of this discovery was made thanks to NIH and/or NSF funding from the USA, or the NIHR in the UK? I don't ask to strictly bring up politics, but instead to try and address the broad lack of understanding of how medical breakthroughs like this are made. It's not done just by drug companies. The article says: > UniQure says it will apply for a licence in the US in the first quarter of 2026 with the aim of laun…

I think it is a UK study. It actually has been going on for a number of years, I've seen one of the PIs giving a number of big conference talks (Tabrizi) for a while

Re: Huntington's disease treated for first time

#56

"If one of your parents has Huntington's disease, there's a 50% chance that you will inherit the altered gene and will eventually develop Huntington's too." Have they never heard of genetic diagnostics? For example with a combination of preimplantation generic testing and in-vitro fertilization you can prevent passing on known genetic mutations to the next generation.

While technically possible, that option isn’t really available to most people. Tho I suppose 17 hour brain surgery with gene therapy is also not available to most people, so fair enough.

Anyway, they’re just describing the heritability of the disease there.

Re: Huntington's disease treated for first time

#57
post #24

This is off topic, slightly but I think a good place to say this: I wish the media outlets would mention the fact that at least one of the scientists in this post is an immigrant in the UK. (in this case I’m not sure 1st or 2nd gen) In the current climate of anti-immigrantion rhetoric around the world, simple things like that might help a little with the perception of immigrants as freeloaders. Just a thought.

This seems helpful, but I think the misattribution of a general "anti-immigrant" sentiment to immigration detractors is part of the problem. Very few detractors in the west have any issues with highly qualified immigrants occupying scientific or research roles. Being opportunistic with which kind of immigrants one offers as Good is partly what's aggravating the issue. It's a radical kind of dismissiveness and deniali…

"Very few detractors in the west have any issues with highly qualified immigrants occupying scientific or research roles." Laughable, these 'few' reside in the highest seats of power and exist among the 'advisors' to the cabinet.

"It's a radical kind of dismissiveness" People keep telling me I'm wrong in the wrong way, if they were nicer I'd consider their opinions. This is an irrational position to stake out, it should go without saying.

"The broad western detraction against immigration at the moment is targeted at specific waves of mass immigration with specific compositions that have specific effects on the places those immigrants have landed."

This is such a laborious way to say "poor, brown or both". Do you get tired of dancing around like this?

"People are primarily concerned about the ability of state, social and corporate institutions"

If you were actually "concerned", you'd advocate for punishing institutions that use cheap (or 'free' if you steal their passport) immigrant labor, rather than targeting those that arrived to fill those positions. Do you have an understanding of why this never happens?

It goes without saying that much of the 'specific immigrant labor' you despise is used to fill the welfare obligations related to care-taking that you are also deeply concerned about.

"dismissive to these concerns" I was assured that 'tone policing' was the domain of the 'woke', yet here it is a second time in the same comment.

"uncontroversial and obvious." You might want to speak to your fellow travelers about which are 'good' immigrants and which are 'bad'. It suffices to say that if they agree in general, but disagree about which group is good, then it is not actually 'uncontroversial'.

Re: Huntington's disease treated for first time

#58

Earlier quoted context omitted.

> Scientists need better messaging or else we're going to stop having breakthroughs like this Sure, but it's really sad that scientists need to justify their funding to the public - they already spend so much time justifying it to the NIH and others for funding. So many people have had their careers jeopardized by finding pulled mid-project. I am really concerned about our research pipeline, because my post-doc frien…

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> No, it's not sad that you need to justify the use of public money.

You don't ask your plumber which computer network you should build for a fortune 500 company for the same reason I don't ask a computer programmer how to fix leaky pipes. People who study in an area actually have much stronger basis for having opinions rather than keyboard warriors who are upset that there mythological studies have been debunked time and time again. That's not science that is cult behavior. If you want to influence that decision process work in that field and provide justifications for that vaccine research and some new angle that's been missed. The American public is not a intelligent member of the medical community there opinion should not have the same weight in day to day operations as the medical community. They can allocate an amount we want to do research and should ask the NIH to do board research because that is effective and has an overwhelmingly strong record having done the background research for basically ever medical advancement made for a long long time.

So, no I think we should allow a panel of experts to evaluate what is worth funding in research. Give the NIH a budget to hire a panel of field experts like they have been for a long time and fund research that panel says is worth it. Autism and vaccine linkage is studied and has been shown several times to have no strong correlation with vaccination. The idea we need to study that more is stupid and experts say it is stupid because it steals funding from actual research into other environmental factors that haven't been studied yet. Maybe its PFAS chemicals maybe its something else.

Re: Huntington's disease treated for first time

#59
post #26

This needs to be said, with Trump's cutting of College Basic Research Funds, many of these great breakthroughs will occur in other Countries. The US is/was the lead in biotech, it is now giving up its lead. That means one of the US largest industries, employing many with high pay, will shrink, due to US policies. In a few years it may not exist unless funding is restored soon. I personally know people who's grants ha…

There’s another connection here, between trump and huntingtons.

Folk singer Woody Guthrie wrote the song “old man trump” about fred trump (donny’s dad), while suffering from huntingtons and living in a building owned by fred.

Re: Huntington's disease treated for first time

#60

More information on the approach: https://www.uniqure.com/programs-pipeline/huntingtons-diseas... > AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE™ silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT) and the actual announcement: https://uniqure.gcs-web.com/news-…

Thanks for sharing this information. Do you (or anyone else here) know if these trends might be expected based on how the treatment works? For example, given than the treatment is only injected into certain parts of the brain, could we expect that some aspects of the disease will be treated better than others?
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