191–200 of 570 posts
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I didn't want to bring up specifics but I'd be lying if it wasn't on my mind.
It would probably be good if more of us brought up specifics more often.
> To accomplish that feat, the treatment is wrapped in fatty lipid molecules to protect it from degradation in the blood on its way to the liver, where the edit will be made. Inside the lipids are instructions that command the cells to produce an enzyme that edits the gene. They also carry a molecular GPS — CRISPR — which was altered to crawl along a person’s DNA until it finds the exact DNA letter that needs to be c…
> But KJ’s treatment — which built on decades of federally funded research — offers a new path for companies to develop personalized treatments without going through years of expensive development and testing. Really incredible story and I'd love to know the process for receiving this, for example FDA approval etc. It's nice to see such in-your-face results from Federal funding programs. Without being political, it's…
I'm not an expert, but I have learned that FDA approval is not actually necessary for treatments and drugs. Your doctor has a lot of leeway when it comes to treatment but she of course experiences more risk of accusations of malpractice when prescribing off label drugs or unapproved treatments. insurance will also rarely cover treatment that is not FDA approved. the requirement for FDA approval generally has more to…
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Now imagine DOGE team of experts cutting this a couple of years ago
I didn't want to bring up specifics but I'd be lying if it wasn't on my mind.
"The implications of the treatment go far beyond treating KJ, said Dr. Peter Marks, who was the Food and Drug Administration official overseeing gene-therapy regulation until he recently resigned over disagreements with Robert F. Kennedy Jr., the secretary of health and human services."
"But KJ’s treatment — which built on decades of federally funded research"
"The result “is a triumph for the American peoples’ investment in biomedical research,” Dr. Urnov said."
"The researchers emphasized the role government funding played in the development."
"The work, they said, began decades ago with federal funding for basic research on bacterial immune systems. That led eventually, with more federal support, to the discovery of CRISPR. Federal investment in sequencing the human genome made it possible to identify KJ’s mutation. U.S. funding supported Dr. Liu’s lab and its editing discovery. A federal program to study gene editing supported Dr. Musunuru’s research. Going along in parallel was federally funded work that led to an understanding of KJ’s disease."
"“I don’t think this could have happened in any country other than the U.S.,” Dr. Urnov said."
This is an article about federal funding of medical research with a cute baby as the human interest bit.
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I suppose a downside (depending on your perspective) of this is that it will make people who are genetically modified in this fashion trivial to detect. That's good if your goals are to detect genetic modification which may be considered cheating in competitive sports. That's bad if your goals are to detect genetically modified people and discriminate against them. I see a near future where the kind of people who loa…
Careful with qualifiers there. I genuinely believe that vaccines can spread illness to the non-vaccinated, since it has happened many times and is well-documented. For example, it's why only the inactivated (aka "dead" virus) polio vaccine has been used in the US since 2000. I'm not arguing about whether the risks of the attenuated virus outweigh the benefits. I think the data are very clear there. (Heh -- and I'm su…
Nothing in medicine is certain. Nearly any medical treatment has a small chance it could kill you. There’s a small, but non-zero chance of a lethal infection even if they injected you with saline, odds that rise dramatically in less than sanitary conditions.
Ironically the use of the attenuated oral vaccine for polio was because of the risk of infection in places where the availability of sterile syringes was hard to guarantee. It’s all about the relative odds.
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> That is one of the most incredible things I have ever read. This is even more great reading behind the above: https://en.wikipedia.org/wiki/Jennifer_Doudna
Walter Isaacson's book "The Code Breaker" is about this subject. I couldn't put it down.
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Edgy! No one has ever considered the mortality of their children ever, or contemplated the difference between death before and after the realization of potential. Wow!
Genuinely don't know why this is edgy. I was trying to understand his logic
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A rare case where the list of awards she's received is so long it needs a separate Wikipedia page https://en.wikipedia.org/wiki/List_of_awards_and_honors_rece...
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https://www.lbl.gov/people/excellence/nobelists/jennifer-dou...
The fact that this treatment "built on decades of federally funded research" is the scary part, given that such funding may disappear.