Crispr Gene-Editing Drugs Show Promise In Preliminary Study 30
Intellia Therapeutics reported encouraging early-stage study results for its Crispr gene-editing treatments, the latest sign that the pathbreaking technology could result in commercially available drugs in the coming years. The Wall Street Journal reports: Intellia said Friday that one of its treatments, code-named NTLA-2002, significantly reduced levels of a protein that causes periodic attacks of swelling in six patients with a rare genetic disease called hereditary angioedema, or HAE. In a separate study building on previously released trial data, Intellia's treatment NTLA-2001 reduced a disease-causing protein by more than 90% in 12 people with transthyretin-mediated amyloidosis cardiomyopathy, or ATTR-CM, a genetic disease that can lead to heart failure.
Despite the positive results, questions remain about whether therapies based on Crispr will work safely and effectively, analysts said. Intellia's latest studies involved a small number of patients, and were disclosed in news releases and haven't been published in a peer-reviewed journal. The NTLA-2002 study results were presented at the Bradykinin Symposium in Berlin, a medical meeting focused on angioedema. The data came from small, so-called Phase 1 studies conducted in New Zealand and the U.K. that didn't include control groups. Results from such early studies can be unreliable predictors of a drug's safety and effectiveness once the compound is tested in larger numbers of patients. The findings, nevertheless, add to preliminary but promising evidence of the potential for drugs based on the gene-editing technology. Last year, Intellia said that NTLA-2001 reduced the disease-causing protein involved in ATTR patients.
Despite the positive results, questions remain about whether therapies based on Crispr will work safely and effectively, analysts said. Intellia's latest studies involved a small number of patients, and were disclosed in news releases and haven't been published in a peer-reviewed journal. The NTLA-2002 study results were presented at the Bradykinin Symposium in Berlin, a medical meeting focused on angioedema. The data came from small, so-called Phase 1 studies conducted in New Zealand and the U.K. that didn't include control groups. Results from such early studies can be unreliable predictors of a drug's safety and effectiveness once the compound is tested in larger numbers of patients. The findings, nevertheless, add to preliminary but promising evidence of the potential for drugs based on the gene-editing technology. Last year, Intellia said that NTLA-2001 reduced the disease-causing protein involved in ATTR patients.
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Just one minor edit could cause cancer
You can also get cancer from chemotherapy and radiation treatments.
If you are dying of cancer, then a 1% chance of getting cancer from the treatment is not a big concern.
or a prion or prion protein to be generated.
There is no plausible way for that to happen. Sure, it is possible, but only in the same sense that a random cosmic ray could also cause a mutation. Cosmic rays cause billions of mutations every day.
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Don't forget the damage caused by that potent oxidising agent, oxygen.
People seem to have this odd idea that it might be possible to live forever. We don't know that that is impossible, but we do know that genes have evolved to control their reproducing machinery (us) to maximise the number of [this particular set of] genes in the next generation. Our genes have no interest, per se, in the the fate of
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The long term effects of any treatment that has not been around long term are impossible to predict.
But the fact is medicine has had constant progress due to new medications and medical techniques being taken into use and that has saved a lot of lives over the years. That is even when some of the things have later been shown to be dangerous to some people. So overall the net benefit has been a HUGE positive despite the occasional problems.
5 replies. 3 of them are nutters. (Score:2)
3 state clear nonsense, 1 is someone replying to point out stupidity and 1 is a swastika.
What happened to Slashdot ?
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Happened? It has always been like this..
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Happened? It has always been like this..
Twenty years ago, Slashdot had way more readers. Most stories had hundreds of posts, and some had thousands. Since then, many people have drifted away, and few new people have joined.
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What gives ?!
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5 replies so far.
3 state clear nonsense, 1 is someone replying to point out stupidity and 1 is a swastika.
Now you see how the moderation system went to hell. And in society as a whole, Uncle Chuck is selecting the population. Those who won't vaccinate and those who refuse new medical treatments are being selected out of the population. Inevitably, sanity will return.
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(*) Based on your sig. Sorry.
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Do you have some reason to think that the normal state for the world is some form of paradise?
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Fantastic news! (Score:3)
This is exactly the kind of development we need. Treating people today with genetic defects is one thing but I choose to look to it's potential where a pregnancy screening that identifies known genetic defects results in a simple prescription to correct the defects before the child is ever born. Would could radically reduce the number of people that have to live with a disability.
On the amusing side, I look forward to the possibility of a white supremacist having their skin turn a nice shade of Mocha and their hair black and stiff as part of their sentence for committing a hate crime.
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Repairing genes is (relatively) easy compared to replacing biological structures they've already built, so correcting a genetic issue when there are as few cells as possible so those structures are built correctly in the first place is ideal.
What you really want to do is have prospective parents take a DNA test, identify any serious genetic problems, and then repair them in the gonads so any children the parents produce are protected against inheriting them.
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This is an excellent point! Fixing the issue before it can even become an issue is an even better approach. However, there are still lots of unexpected pregnancies that would benefit from in vivo treatment as well.
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In this future I see limbs regrown nerves refused or grown. All parts specific and general. I've heard of re
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You'd have to introduce the whole biochemical equipment of metamorphosis into the vertebrate genome. That's a huge project. It's probably be a lot easier to get people to grow a spare arm, leg and head each.
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So you think it's punishing someone to give them dark skin and hair? Isn't *that* racist itself?
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So you think it's punishing someone to give them dark skin and hair? Isn't *that* racist itself?
Well there's a dumb hot take. The point is to make them live as the very thing they hate. It's not necessarily a punishment either as it could change their perspective.
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The reagents they require cost a lot more. Plus, of course, you need the skills to do the lab work required to get the correct reagents into the correct sets of test tubes at the correct time. That's a relatively simple path - a few thousand hours in the lab would suffice.
There's a reason these machines are run by technicians who are MSc or PhD level.