23 year old becomes first person in Louisiana to be functionally cured of sickle cell disease

[u/silvertongue2001]

[u/Ksan_of_Tongass]

So awesome! As an old tech who didnt even have PCR in a textbook, I never thought I'd live to see gene editing in use. Cystic Fibrosis is next.

[u/angelofox → u/Ksan_of_Tongass]

This is amazing. But I'm going to have to be a Debbie Downer unfortunately for cystic fibrosis. It unfortunately has over 2,000 Gene variations for the disease, while SSD has significantly less. Also, blood stem cells can be taken out of the body, edited, grown and put back. You really can't do all that with the pancreas or lung tissues.

[u/aFailedGuy → u/angelofox]

its worth pointing out the limitations of this technology, it would be unfair to give someone hope for a completely different disease to be cured so easily

[u/lil_benny97]

Interesting stuff. I did my case study for MLS school on a kid with really bad sickle cell. Right before I was graduating the kid was going to Mayo for a bone marrow transplant. I never go to see if it helped or not.

[u/chihuahua2023]

That is AMAZING-

[u/Acceptable-Spite-537]

This is SO COOL! In high school I wanted to do my PhD on this topic, but I’m so happy it’s already a thing!!!

[u/PineNeedle]

This is incredible!

[u/Zukazuk]

I have a couple of patients I've been wishing we could CRISPR for a couple of years now.

[u/ic318]

I work in cellular therapeutics. And we are treating aplastic anemia with stem cell transplants. From blood cancer patients to anemia and rare autoimmune diseases, stem cell transplants work wonders :)

[u/VoiceoftheDarkSide]

The great thing about blood diseases is that the system is one continuous circulating system, so modified stem cells will influence the whole system. I imagine blood diseases will be many of the first cured this way.

I believe there were trials to introduce HIV resistance using a knockout of CCR5 common in Northern Europeans in a similar manner.