The Rarest of All Diseases Are Becoming Treatable (msn.com)
- Reference: 0180341857
- News link: https://science.slashdot.org/story/25/12/09/1612202/the-rarest-of-all-diseases-are-becoming-treatable
- Source link: https://www.msn.com/en-us/health/other/the-rarest-of-all-diseases-are-becoming-treatable/ar-AA1RW6se
The treatment's significance extends beyond one child. Scientists at UC Berkeley's Innovative Genomics Institute and the Children's Hospital of Philadelphia are now planning clinical trials that would use Muldoon's therapy as a template, tweaking the molecular "address" in the CRISPR system to target different mutations in other children with urea-cycle disorders. Last month, FDA officials Marty Makary and Vinay Prasad announced a new drug pathway designed to accelerate approvals for such personalized treatments -- a framework inspired in large part by Muldoon's case. Current gene-editing delivery mechanisms limit treatments to disorders in the blood and liver. Many families will still go without bespoke therapies.
[1] https://www.msn.com/en-us/health/other/the-rarest-of-all-diseases-are-becoming-treatable/ar-AA1RW6se
You know rich people are (Score:3)
...going to fly to CRISPR Island and have their genes edited for a bigger dick, longer jiz, bigger tits, etc.
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Sure, plus faster reflexes, bigger muscles, maybe cat ears and tail. The wealthy will always get a disproportionate share of the pie. But as long as those who need this tech can get it (which includes both "availability" and "affordability"), that's okay.
There is a danger of a Gattaca world, but there is always a danger of a dystopia. I could wish that fewer Americans would be cheerfully voting for dystopia to "own the libs" or "stop woke" or something moronic like that, but whatever.
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What's your point. Once upon a time the wealthy were installing indoor plumbing and fancy water closets while everyone else was still using outhouses or chamber pots. They subsidized the capital expenses necessary to for various businesses to offer their goods and services and refine the invention to make it better, less expensive, and more widely available. It was the same for automobiles, computers, and just about anything else you might care to name. Why would you expect it to be any different here?
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I mean, sure, we could go right to THE INFERIOR HUMAN MUST NOT REPRODUCE (funny how the speaker always puts themselves in the superior category, even though such statements demonstrate a inferior mental capacity). Or, plan B, we could fix those major genetic problems so that inherited diseases are not longer inherited (and, even if they are inherited, they are no longer a problem).
Crohn's please (Score:2)
Cure Crohn's please
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There's people with Crohn's who have gone on the carnivore diet and said they were able to stop getting Remicade. I don't have Crohn's so I can say how true those claims are, but I'll say it's worth looking into. Theoretically since it's immune related, hypnosis should have an effect on it as well, but I haven't researched that angle. Hypnosis is oddly good at dealing with immune related issues, you can cure your allergies using it.
(I have a relative with Chron's. She's not interested in trying carnivor
Meanwhile... (Score:3)
Meanwhile, the most preventable of diseases are becoming commonplace.
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This is a self-correcting problem for any detrimental behavior in the long term. I know it tears some people up inside, but let others make their own decisions freely and allow them to deal with the consequences of them. You can't reason someone out of a position that they did not use reason to arrive at in the first place. Sure, some people will never learn to quit touching the stove, but many other people can learn from their misfortune.
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It's only self correcting when the signs are highly visible and visible before the infectious period starts. For an example, I won't be surprised if HIV rates start going back up despite us being able to control transmission now.
Soon to be killed (Score:2)
It will be anti-christian, witchcraft and a very dangerous vaccine.
If you can not pray it away, it is EVIL /S
Just wait until they decide it interferes with "gods plan" and that THEY have the right to control YOUR life..... just like women healthcare .
Science moving forward...country moving backward (Score:3)
It's very nice to see more and more illnesses being treatable with advancements in science and also disheartening to see others trying to take your country back to the middle ages.
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On what do you base this idiotic statement? Don't you know, according to DementedDon, that Dems are "terrorists"?
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lol what are you even talking about?
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RFK Jr. is a nutjob. When you are spiraling in conspiracy mental breakdown, party becomes more vague. Democrats have nutjobs (which used to include Mr Brainworm), but democrats have this habit of not giving nutjobs any power or position, not even Assistant Dog Catcher. Republicans, however. have welcomed nutjobs with open wallets.
So, on the one hand, there are just as many "liberal" nutjobs as "conservative" nutjobs. But since republicans are happily giving them cushy government jobs with power over hea
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One of the major advances, [1]outlined in this Wired story [wired.com], involves personalized creation of mRNA vaccines to treat cancer
This is made possible by identifying differences between the cancer, and the person's normal DNA, then creating a mRNA vaccine that tunes the body's immune system to attack the cancer.
Unfortunately, this requires a regulatory system that recognizes the validity and safety of mRNA vaccines in order for it to be covered by insurance
The path we are on right now, will ONLY ensure this treatmen
[1] https://www.wired.com/story/wired-health-lennard-lee-cancer-vaccines/
Re: Science moving forward...country moving backwa (Score:2)
Until there is some super-automated AI pipeline to identify novel neoantigens purely from cancer DNA with no human needed in any of the millions or billions of required steps, truly personalized mRNA vaccines seem intractable to me. Identifying antigens on the outside of the cancer cell from DNA alone is very difficult indeed.
The only tractable personalized vaccine in the short term is much much simpler, just use the tumor cells and forget about the DNA.
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Bullshit, at best they work the other way around. Identify antigens on cancer cells and try to work back to DNA.
Lets assume you do it the right way around so it doesn't require science-fiction, who's selling or even developing the machine which can do that for a single patient in reasonable time? Using many millions worth of general purpose machines for months with phd students babysitting the process, is not tractable.
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In conclusion, no one is even developing a machine which would make identifying the RNA sequences encoding cancer antigens tractable.
Meanwhile many labs can implement the protocols for tumor tissue based vaccines at negligible cost, that's the low hanging fruit for cancer vaccines. RNA approaches are decades out, identifying antigens from DNA alone sci-fi at the moment.
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There's no reason for this to be expensive. I've made CRISPR nanoparticles and mRNA vaccines for research purposes. I can tell you the process is simple enough that a person with good financial means can make these himself in a garage with below $150k of capital expenditure. Note .. it can be done for under $60k too in a crude manner. I put $150k because you'd want a machine called an FPLC for purification purposes, freezers, and maybe a DLS. Now before that asshole who always argues with me shows up, it's
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> There's no reason for this to be expensive.
Based solely on material and operational costs, most drugs should be much cheaper than they are. The big difference is the profit motive in the absence of a truly free market. If there were sufficient suppliers who were free to compete by lowering prices, then prices might be reasonable or affordable. However, often suppliers with either government connections or who can erect other market barriers can prevent competition. This is the paradox of competition, that competition often leads to actions to in
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One simple trick: don't call it a vaccine. Call it an immune system booster. Bonus points: formulate it with a little bit of stearic acid (beef tallow) in with the other lipids.
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> Science moving forward
I don't know if I'd call giving up on prevention or cures and focusing on treatment "moving forward" - I'd call it profitable .