Mechanism explained- CRISPR Drug for Sickle Cell Anemia (Casgevy)
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- Опубликовано: 5 авг 2024
- 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!
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.
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.
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.
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.
Didn’t even know about this happening, so I’m glad you made a video
concise and thorough explanation - great job!
Brilliantly explained. Thank you for the education. Great job
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.....
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.
Very clear explanation. Thank you!!
you've done an incredible job of explaning tyy
Very interesting and I’d love to learn more.
Well explained. Thank you
Thank you!! Appreciate it! You are the best. Big huge thank you!
Thank you so much!!
Very well explained. It's very clear to me now. Thank you so much. Although, some graphic content would be appreciated.
You are the best, thank you
People with sickle cell trait has mild symptoms, but with sickle cells disease, they need this medication.
Thanks for sharing...🥂
Well done
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
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.
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😅
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 ❤❤🍀🍀❤❤❤❤❤
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Cure autism with crispr.
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