America's First CRISPR Trial is Still Nearly 100% Effective 3 Years On (newatlas.com) 49
Incredible new data presented recently at the European Hematology Association Congress has revealed an experimental CRISPR gene editing therapy is both safe and effective up to three years after treatment. The follow-up results come from one of the longest-running human trials using CRISPR technology to treat a pair of rare genetic blood diseases. From a report: The first human trial in the United States to test CRISPR gene editing technology started back in 2019. The trial focused on two rare blood diseases: beta-thalassemia and sickle cell disease. The treatment involves first gathering stem cells from a patient's blood. Using CRISPR technology a single genetic change is made, designed to raise levels of fetal hemoglobin in red blood cells. The stem cells are then re-administered into the patients. Initial results were extraordinarily promising. The first two patients treated were essentially cured within months, but questions over long-term efficacy remained.
A follow-up announcement last year continued the impressive results with 22 patients treated and all demonstrating 100 percent success. Importantly, seven of those patients were 12 months past the initial treatment with no waning of efficacy. Now, a new data release is offering results from 75 patients treated with the groundbreaking CRISPR therapy, now dubbed exa-cel (exagamglogene autotemcel). Of those 75 patients treated, 44 were suffering transfusion-dependent beta thalassemia (TDT) and 31 had severe sickle cell disease (SCD). All but two of the 44 patients with TDT were essentially cured of their disease, needing no more blood transfusions. The two TDT patients still requiring blood transfusions had 75 percent and 89 percent reductions in transfusion volumes respectively. All 31 SCD patients were also free of disease symptoms at long-term follow-up.
A follow-up announcement last year continued the impressive results with 22 patients treated and all demonstrating 100 percent success. Importantly, seven of those patients were 12 months past the initial treatment with no waning of efficacy. Now, a new data release is offering results from 75 patients treated with the groundbreaking CRISPR therapy, now dubbed exa-cel (exagamglogene autotemcel). Of those 75 patients treated, 44 were suffering transfusion-dependent beta thalassemia (TDT) and 31 had severe sickle cell disease (SCD). All but two of the 44 patients with TDT were essentially cured of their disease, needing no more blood transfusions. The two TDT patients still requiring blood transfusions had 75 percent and 89 percent reductions in transfusion volumes respectively. All 31 SCD patients were also free of disease symptoms at long-term follow-up.
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Some will be OK with it, some won't.
As long as it's not forced on anyone, I don't see a problem.
Re:People weren't OK with mRNA vaccines` (Score:5, Insightful)
When people are given a severe diagnosis a lot of "principles" go out the window.
How many stories of people about to go on ventilators who were now asking for the vaccine did we hear.
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You think they're going to be OK with this???
They won't, but who cares? The people who matter are mutation disease patients who do take the treatment.
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Frequent blood transfusions are a slow dead, unless you get a blood shock then it can go a little faster.
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Treating a specific disease is different that trying to prevent one. I suppose if you actually had a blood disease then you might want to have it treated.
Re: People weren't OK with mRNA vaccines` (Score:2)
"My penis isn't big enough" disease. Release the genetically enhanced super dongs! Really though, wasn't there some theory that technology like the internet/etc can have a large initial surge if the market is sex?
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"You think they're going to be OK with this???"
That's healthy, stupid people.
Sick, smart people would have no problem not dying.
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I Won't Eat GMO food... (Score:1)
But you can GMO my DNA..
Re:I Won't Eat GMO food... (Score:5, Insightful)
I seek to avoid GMO not because I think the technology is inherently bad, but mostly because I don't trust anyone involved in monetizing it. The primary reasons it's used are to enable the use of additional chemicals, despite all the people who love to claim that farmers want to use as little of them as possible — both things can be true at once.
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And meanwhile, I will not buy any product that is advertised as No GMO. Not because there is anything wrong with the product, but because I don't support anti-scientific messaging.
Re:I Won't Eat GMO food... (Score:5, Insightful)
And meanwhile, I will not buy any product that is advertised as No GMO. Not because there is anything wrong with the product, but because I don't support anti-scientific messaging.
I buy whatever is cheapest because it is 2022 and I have a family to feed. It's generally GMO, but not always.
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Same here, I specifically avoid anything with that stupid label.
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And btw I do think there is something wrong with non-GMO product. It raises the price of food for one thing. Requires larger farms, and increases price per kilo of food. Also, non-GMO doesn't mean pesticide-free .. they usually need more pesticides, a lot more.
You cannot avoid CRISPR if you tried (Score:2)
SARS-2 (Covid-19) was almost certainly the result of CRISPR like technologies to add the Furin Cleavage Site to the spike protein. The virus finds you!
It is nice that CRISPR helped some people, but it would need to help a hell of a lot to make up the damage of Covid-19.
GMO crops can also cause issues. Round up ready wheat is resistant to Glyphosphate, the main herbicide used, which makes it easy for farmers to deal with weeds -- just spray the lot. Trouble is, those synthetic genes have managed to get in
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Why would you need CRISPR to create that furin cleavage site when there are other methods that are 100x easier? Get a clue.
Re: You cannot avoid CRISPR if you tried (Score:1)
I know you don't believe in PPE, i.e masks, gloves, etc, but the reason you got sick is because you take a spike protein in your butt from random strangers. Please, for everyone's sake, use a condom already.
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I seek to avoid GMO not because I think the technology is inherently bad, but mostly because I don't trust anyone involved in monetizing it.
Because it's so profitable to kill your customers.
Re: I Won't Eat GMO food... (Score:1)
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If it killed them in a trivially provable way, or rapidly, then they wouldn't do it.
The world is replete with examples of products which are killing us as we use them. Fossil fuels and ICEVs. Fabric softener. Alcohol and tobacco. Plastics in general. I could go on but I'm depressed already.
Re:I Won't Eat GMO food... (Score:4, Funny)
Eating stuff that has DNA in it sounds gross. Ban all foods that have DNA in it.
Re: I Won't Eat GMO food... (Score:2)
This isn't as strange as you'd think. You are essentially petitioning for food only to be sold that's been cooked and predigested a bit.
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Most of it has been treated with DHMO [dhmo.org], though. And you know how dangers that is.
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This isn't as strange as you'd think. You are essentially petitioning for food only to be sold that's been cooked and predigested a bit.
So, a McDonald's hamburger.
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Cooking does nothing to DNA.
Re: I Won't Eat GMO food... (Score:2)
Weird nobody has mentioned it yet. GMO food is made with a "gene gun". They blast a seed with it and mess with it's DNA like using a shotgun for CRISPR. Maybe it work, maybe it dies, maybe something fun and interesting. CRISPR is much more targeted. In both cases, we have little understanding of any long term effects.
https://www.sciencedirect.com/... [sciencedirect.com]
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GMO food is developed in a variety of ways, including CRISPR.
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Huh? Fuck off. Gene therapy almost got permanent-banned because of an accident around 20 years ago, so everyone is very cautious. Basically the patient's immune system said a literal "fuck this" and went ballistic attacking every organ in his body simultaneously. You can't have it where one accident nearly shuts down an entire field and then ask stupid questions like "where are the cures?" Fuck off. Right now it costs a billion (with a B) dollars to be risked to get something into clinical trial, no invest
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So far, so good... (Score:1)
"Incredible new data presented recently at the European Hematology Association Congress has revealed an experimental CRISPR gene editing therapy is both safe and effective up to three years after treatment".
Well, that just leaves a few decades to go in the average life expectancy.
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Shame about the need for chemo (Score:2)
Killing the existing bone marrow with chemo is a risky procedure. Where's the viral gene editing cures?
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Is that what they did here? TFA doesn't say.
I knew a woman who had an autologous bone marrow transplant, and it seemed extremely painful and dangerous. It wasn't a cure for any disease - it was a cure for the massive amount of chemo they gave to kill her stage 4 breast cancer. The idea was to harvest her bone marrow, give her enough chemo to kill her, or at least kill her bone marrow, filter out any cancerous cells from the harvested bone
R&D funding in med is a conundrum. (Score:2)
Humanity somehow cured widespread diseases repeatedly in the span of a couple of generations, but ever since then medical science has become an obscurantist shadow world of ambiguous treatments, politician's stat games, and (at least psychologically on the part of the lay public) a return to Early Modern skepticism at the basic competence of practitioners.
T
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"Humanity somehow cured widespread diseases repeatedly in the span of a couple of generations,"
so the low hanging fruit has already been picked...those naughty scientists.
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Exactly, once vaccines became a thing it was pretty quick to adapt them to the most common viral diseases. Thing is they only work on those viral diseases.
Antibiotics are the same thing, covered a huge amount of common illnesses, but they only apply to bacterial/fungal type infections.
Now we are left with the tricky bastards. Cancer is just our own cells going haywire, much tougher of a problem to solve than an external threat.
Hand-wavy excuses are not useful. (Score:2)
Two things seem to be at odds: Our level of biological knowledge is far beyond the early 20th century's, and yet the pace of progress in applying it appears
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Why is it always a "rare" condition? (Score:2)
Why do these breakthroughs always seem to treat some rare condition instead of a common one?