Medical progress has been insane in the last few years through technological breakthroughs. It's not out of reach to think that most types of cancers will be curable 20 years from now on.
Huntington's disease treated for first time
21–30 of 133 posts
Re: Huntington's disease treated for first time
#22Re: Huntington's disease treated for first time
#23More 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-…
Why does it have to be delivered through brain surgery?
The brain-blood barrier typically only allows small, non-polar molecules to pass through into the brain, which complicates a lot of neuro/psych treatments.
Re: Huntington's disease treated for first time
#24This 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.
Re: Huntington's disease treated for first time
#25Earlier quoted context omitted.
Why does it have to be delivered through brain surgery?
The major hurdle of current gene therapies is delivery to the tissue where the defective gene product is causing damage. For instance lipid nanoparticles are only being used to deliver gene therapies to the liver, because if you inject them they just end up there and not much anywhere else. In this case they are using an virus called "adeno asociated virus 5" (AAV5), which does not naturally infect the brain AFAIK. T…
I guess it needs to get across the blood-brain barrier. But that shouldn’t take 10+ hours of surgery, I don’t think.
Re: Huntington's disease treated for first time
#26This 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 have been cancelled due to these cuts.
But, glad to see other Countries funding their research. I wonder in face of one of the largest blunders made by the US, are they increasing funds ?
Re: Huntington's disease treated for first time
#27What 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'm pretty sure some of it is direct funding by governmental agencies, but even if that wasn't the case, all the basis of the theory, and the groundwork was laid by researchers and universities using those grants. You need public money for a healthy society
Re: Huntington's disease treated for first time
#28Medical progress has been insane in the last few years through technological breakthroughs. It's not out of reach to think that most types of cancers will be curable 20 years from now on.
[flagged]
Re: Huntington's disease treated for first time
#29Earlier quoted context omitted.
I’m assuming the viral vector can’t pass the blood-brain barrier.
But it doesn’t take 10+ hours to surgically get a virus across the blood-brain barrier, right?
The video specifies that the drug is infused over 8-10 hours. Probe placement - again, as depicted in the video, because I don’t see a real methods section - should take about 1-2 hours. The video isn’t clear if this is interactive MRI or just a preop scan that is then loaded into a stereotactic navigation system in a regular operating room, but the former would add another hour at least. MRI is not fast.
Re: Huntington's disease treated for first time
#30Medical progress has been insane in the last few years through technological breakthroughs. It's not out of reach to think that most types of cancers will be curable 20 years from now on.
I think its like WW2, loads of amazing tech came from war. Covid was a bit like that for bioTech.