New CRISPR-based sickle cell treatment, explained

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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[techno]

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

[IRONMAN]

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

[moMoeXe]

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.

[GraceFidel]

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

[josephmoses]

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.

[THX-wcjo]

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

[leilaniLA]

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

[vakudibeardefender]

Science fiction is becoming technology facts.

[RAMESHKAFLE-yc]

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

[M.m]

And somehow the stock crashes after approval

[giakolou]

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

[hershekissed]

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

[hershekissed]

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?