UK approves gene therapy to cure two blood disorders - BBC News

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In a world first, medical regulators in the UK have approved a gene therapy that aims to cure sickle cell disease and beta thalassemia.

The treatment is the first to be licensed using the gene-editing tool known as Crispr, for which its discoverers were awarded the Nobel prize in 2020.

It is a revolutionary advance for two inherited blood conditions, both triggered by errors in the gene for haemoglobin.

In trials, 28 out of 29 sickle cell patients were free of severe pain and 39 of 42 beta thalassemia patients no longer needed blood transfusions for at least a year.

The trials are continuing in the UK, US, France, Germany and Italy.

#GeneTherapy #SickleCellDisease #BetaThalassemia #BBCNews
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Actually something great of a public acknowledgment of the years of many people's time, hard work and energy spent, studying, researching and properly analysing this health condition. It is a very useful health tool for the sickle cell affected people and finally they are getting a proper cure for their health situation after years of waiting for it.Congratulations.

vj.joseph
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I'm very happy for the people suffering from Sickle Cell. I feel the need to express my own experience with TWO chronic illnesses that have ZERO treatments and virtually no support. I've suffered from one since I was 8, and the second being Long Covid. I'm mostly bedridden at this point and I have suffered every day for half a century. The worst part of no treatment or support is not only unrelieved suffering and disability--it's the despair brought on by lack of hope and help, because there are no treatments or research offering hope for the future, or services and support to give you a hand surviving each day. The only thing I was told by the medical and social professionals I consulted was a Facebook page fellow sufferers had created on their own. I don't do Facebook and after seeking help for fifty years for an illness ignored, I know it only backfires and hurts all the more. It's hard for me to hear "we only had a handful of treatments before this cure." Not because people suffering from Sickle Cell do not deserve this, of course they do. But because sufferers of other illnesses do not deserve to be abandoned in a desert of despair. Please pay attention to orphan illnesses and Long Covid.

MicaOShea-oeir
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These are the kind of news we need ❤❤❤🥰🙏🏻🙏🏻🙏🏻

camillac.s.
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Thank you scientists and researchers!!! This is great news! 👏👏👏

catalinacurio
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Thanks for the research work and the progress made so far!

Cuzibor
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I have Asherson's disease, AKA Sludge Blood, #289 to ever be diagnosed. It also causes mishappen cells and lowers hemoglobin and iron saturation within the RBCs. I take iron infusions because the greatest irony pun is that I'm allergic to oral iron supplements.

Very intrigued by this development and wondering if it would help me.

😒💀

DarcyHocker
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This is such a great innovation. Kudos to all involved 👏🏾

rinretdashan
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This is great news, I've seen the pain this inflicts on people, hopefully no more.

wannabeadrummer
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That's great news! Science is really progressing.

onenation
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They approved it… but won’t approve it for treatment on the NHS.

Kizzy
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Incredible. Great. Britain is now leading innovations. Thanks to acceptance of brilliant minds from all over the world.

efeoba
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I'm 32, is it too late for me to get the treatment? I need to rid myself of this horrible disease

tashakashito
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During the pandemic it was noticed that different ethnic groups had different reactions to the virus. It's similar with sickle cell anemia, when different groups have different outcomes in the NHS it isn't necessarily because of discrimination.

sebastianguerre
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I knlw i had enemia one time i cooked clam juice and clams * i minced them and you have to like cheese i made clam chowder
and added a jar of alfredo sauce also ..

the other way is to use vegetable broth clam juice and clams * salt pepper onion garlic salt & pepper.. i went back and my anemia levels were perfect .. clam.soup chowder with alfredo sauce and vegetable broth sauce and drank
Tomato v -8 juice .

I know its diferent .sickle cell anemia yet it is anemia it will not hurt to try and it was less then 7 days . Of esting this every other day i dont know every other day & regular foods every other day then 1 bowl friday or sat or sunday and then no more the rest of the week ..or one time a week.

dennaspradlin
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Reporters have a green background. 😂😂

Alorpothojatri
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Ma'am my cousin suffering from thalassemia would like help him

AbuShaikh-zt
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So how long does it take to cure? She didn't answer. Am curious

DamZFXBeats
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Sickle cell disease affects mostly people who are poor and should not have children to begin with

phoque
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How much did they say it costs please ?

the
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My son is 4 years old and he has thalassemia major. I want to undergo gene therapy treatment. When will this treatment be available in India? please inform me

anantakumar