Mechanism explained- CRISPR Drug for Sickle Cell Anemia (Casgevy)

I've heard a lot of people talking about this drug for sickle cell anemia but had a super hard time finding out the actual mechanism of how it works. So here is a high-level overview of everything you need to know to understand the basics of the first-ever CRISPR-based drug, enjoy!

[@mahes303]

First time it is clear to me how this technology works and what good it can do for people with this terrible disease. Thanks for the clear explanation.

[@sahwahhammad5219]

The explanation was very clear and easy to digest for both medical and those outside medical field. Regarding Sickle cell disease, it is best described as a missense mutation rather than SNPs because although it is a single nucleotide change it exists in less than 1% of population and it is associated with disease while SNPs are mostly non disease causing and exists in more than 1% of population.

[@TheLabMouse]

Thanks for the clarification! I had actually been taught about SNPs via sickle cell specifically. Likely since it is disease causing so it's easier to teach and resonates more. But you are right, nowadays I mainly hear the term SNPs thrown around to describe benign and nocoding regions.

[@RockerProf]

Agreed! MTHFR can have SNPs. They naturally occur in about 40% of the population, and while not resulting in optimal proteins, are not technically a disease state. The sickle cell problem is caused by an infrequent missense mutation, one letter, which leads to a wrong amino acid, and being autosomal recessive, results in a disease state. A subtle but important distinction.

[@isabelbrickner136]

Didn’t even know about this happening, so I’m glad you made a video

[@horacefields736]

Very good explanation. I did need to pause the graphics, because there's a lot of information in them, but it's fascinating to see the mechanism.

[@patrickdaoust01]

concise and thorough explanation - great job!

[@user-oh8hu8sm8z]

Very interesting and I’d love to learn more.

[@errolcampbell6985]

On my 58th year with sickle cell anemia...... they have done so much studies about it since I was a child..... I won't even think about any new treatment.....

[@kimquigleybrown]

Brilliantly explained. Thank you for the education. Great job

[@shortfutiball7023]

you've done an incredible job of explaning tyy

[@piyananboonprasirt110]

Very clear explanation. Thank you!!

[@ilovegod0106]

Well explained. Thank you

[@samnoah9451]

We have already 2 million and 4 hundred thousand stems cells and we need 6 hundreds thousands more to make the stem cell transplantation well you pls pray for us to be free from sickell

[@laurasolano1654]

Thank you!! Appreciate it! You are the best. Big huge thank you!

[@liyakhusnullina4310]

Thank you so much!!

[@hadeerdodo823]

You are the best, thank you

[@steveholmes1736]

Well done

[@PAC9322]

Thanks for sharing...🥂

[@user-qn9xx2cv5t]

What happens to the original defect beta-globin gene? Does removing BCL11a alone stop the expression of the beta-globin gene? If not, it seems there may still be issues with blood clotting.

[@marcxu6173]

People with sickle cell trait has mild symptoms, but with sickle cells disease, they need this medication.

[@andreazappacosta2906]

Hi, I was wondering if fetal Hb has an higher affinity to oxygen, wouldn’t this cause problems in oxygen exchange from capillaries to tissues? It would be like having Hb constantly at R state, which can get saturated very fast, but would also be very slow at giving away oxygen. I’m sorry if what I said makes no sense to you, I’m still a student😅

[@evabogatec398]

Cure autism with crispr.

[@Elinondebra]

Meeting GOSTO HEB has really been one of the best day of my life after years of suffering I have been finally cured from cold sore thanks doc. I will always tell the world about your YT profile ❤❤🍀🍀❤❤❤❤❤ . . . . .

[@RizwanUllah-qk1wp]

kindly share your email.