New CRISPR-based sickle cell treatment, explained

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The Food and Drug Administration has approved the world’s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder.
The new medicine, called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics. Its authorization is a scientific triumph for the technology that can efficiently and precisely repair DNA mutations - ushering in a new era of genetic medicines for inherited diseases.
This new video from STAT explains how the drug works, and how it is manufactured.
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Founded in 2015, STAT is a global digital media brand that focuses on delivering fast, deep, and tough-minded journalism about the life sciences industries to over six million monthly site visitors and an additional 20 million readers on the Apple News app. STAT takes you inside academic labs, biotech boardrooms, and political backrooms, casting a critical eye on scientific discoveries, scrutinizing corporate strategies, and chronicling the roiling battles for talent, money, and market share. With an award-winning newsroom, STAT provides indispensable insights and exclusive stories on the technologies, personalities, power brokers, and political forces driving massive changes in the life sciences industry - and a revolution in human health.

Опубликовано:

30 сен 2024

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Комментарии : 55

@7techno77 5 месяцев назад

Essentially it's a bone marrow "transplant" but with patient's own cells instead of donor, so no rejection risk?

@Tallturk 5 месяцев назад

Correct, unless they react to the Cas9 enzyme components (bacterial), but so far no evidence indicating good safety

@vakudibeardefender3953 20 дней назад

Yes. no rejection because its his own DNA.

@MR.Science12 5 месяцев назад

anyone tell me this treatment can be done for thalassemia trait ?//

@Tallturk 5 месяцев назад

Yes, it’s theoretically applicable to any hemoglobinopathies (diseases related to suboptimal hemoglobin function in carrying oxygen)

@MR.Science12 5 месяцев назад

@@Tallturk wow thanks you hope i can one day

@vakudibeardefender3953 20 дней назад

Science fiction is becoming technology facts.

@RAMESHKAFLE-y5c 9 месяцев назад

Good news thank you 😂😂😂😂😂😂❤❤❤❤❤❤❤😂😂😂😂❤❤

@moMoeXe 7 месяцев назад

Me as a "sickle cellian" are happy and sad at once. How are normal people supposed to pay for that? I'm born with it and suffered my whole life 33years. I dont know how much I still got. And a lot of South european and north african(Morocco,Tunisia,Algeria, Egypt - Berber/Amazighs/Touareg & Arabs) & Asian(South East like India,Thailand,Indonesia, Phillipineans ect.) people got it too. I'm born and raised in Berlin Germany and had a lot of different people from different places on the world with me. I heard in India its a big thing tho from a person I once had the pleassure to know. So its not only black people who got it. In my reasearches I found out its all over the World in places where its warm climate. It makes sense. It says we got 7-8million people world wide with sickle cell. But i think the real number is maybe the double of it. Like 15-20mio people worldwide. So its profitable.

@indiancitizen3013 7 месяцев назад

That's true , I'm from India & suffering from sickle cell and as a Indian we can't afford the multi million dollar therapy 😢

@Tallturk 5 месяцев назад

Every innovative new product through history (cars, mobile phones, antibiotics etc) essentially started off as a luxury product that few could afford, then evolved through industrial manufacturing into the products that most can afford given a stable economy and plenty of education

@hershekissed 9 месяцев назад

So how is this going to be used practically? Insurance companies don’t want to pay for treatments like hydroxyurea😂

@ClintStone-t9m 8 месяцев назад

once you have enough people to actually buy such products, it becomes cheaper. Producing target DNA and Gene-Vectors on a large scale is relativly cheaper than doing it on a smale scale, because most of these intermediate steps like producing the Guide-RNA are largely independet of quantity, so the more you can create in one go, the more efficient you are. That said, even if actual material cost and leg work necessary for to produce this stuff and do this therapy on someone, companies obviously would sell it at a much higher price for more return. That's why we need to legalize this stuff, so that pioneer-companies like have competition to drive down the price.

@hershekissed 8 месяцев назад

@@ClintStone-t9m Sickle Cell is a predominately Black, melanated disease. I’m sure there’s enough of us to turn a profit. I do agree however to some kind of open sourcing of the tech. It’s just so disappointing, a cure that no one can access. There’s a generation of kids that have been told the cure would come in their lifetime, they never mentioned it would be inaccessible.

@Tallturk 5 месяцев назад

It’s cheaper in the long run vs cost of lifelong therapy

@saguirre760 4 месяца назад

Sickle cell is an expensive condition to treat. Last sickle patient I had was in the hospital for about three weeks. So in the end insurance companies will save money by paying upfront for the treatment.

@sharktrb Месяц назад

That used to be the question when Tesla first entered the market. You're basically given the chance to invest the bottom and dump it on retail 10 years later

@THX-wc2jo 9 месяцев назад

Standing light behind you isn’t helping your lighting - your face is not well lit and camera exposure is adjusting for the bright light being you

@El-Buddah 24 дня назад

Always a whiner and complainer even at a free show. Please proceed to the nearest exit sir and close the door behind you.

@Tallturk 5 месяцев назад

MaxCyte inside :)

@Ashzxor 4 месяца назад

Can you answer a question. I'm 13 I live in Canada and I have sickle cell. I don't really know how assurance work but is it going to be free for me? Or I gotta pay for it?

@leilaniLA 9 месяцев назад

Who will be the guinea pig and which one can afford?

@loganwoodxyz 9 месяцев назад

FDA approval means it has already been tested. “Guinea pigs” (human beings) have already completed their treatments.

@josephmoses4902 9 месяцев назад

Countless thanks to you Dr Igudia for working out wonders with your Herpes medicine. Your sincerity, honesty and truthfulness are really appreciated. The world needs more doctors like you.

@GraceFidel 9 месяцев назад

We need a someone who has used it to come tell us please

@lifeofdreya1521 8 месяцев назад

I’ve had it done

@GraceFidel 8 месяцев назад

@@lifeofdreya1521 please how was it and what was the cost

@DeborahOkorode 8 месяцев назад

@@lifeofdreya1521stop the cap!. Are you for real?

@ClintStone-t9m 8 месяцев назад

@@lifeofdreya1521 damn, when have you done it and how do you feel? sorry but I'm really curious

@lifeofdreya1521 8 месяцев назад

@@ClintStone-t9m I did it in 2020. It took me a long time to recover because I have other health issues but I’m doing Fantastic now. I moved across the country to live, something I never thought I would be able to do

@M.m475 9 месяцев назад

And somehow the stock crashes after approval

@giakolou2876 6 месяцев назад

Reported for spamming. No useful information, u just pulled 1st summary from Wikipedia. L

@hershekissed 9 месяцев назад

And why not have a Black person explain a Black problem to Black people? Yes I know other ppl of color can get it 🙄 ignorant people say what?

@loganwoodxyz 9 месяцев назад

Go make your own video then. What?

@aavvaallooss2 9 месяцев назад

You Americans are obsessed with the "racial" issue.

@loganwoodxyz 9 месяцев назад

Imagine editing your comment to sound even less mature. 😂

@chris47293 9 месяцев назад

you stand at a moment that has blown the door open on genetic disease theraputics and all you can do is complain about what the person who explains the tech looks like? i hope you find some peace.

@Billy4321able 9 месяцев назад

You're right about insurance not covering this ever, but why do you have to make everything about race issues? Are you American? They seem to do that a lot. I would just be happy any research is going into this disorder at all, and a cure no less!