My Authors
Read all threads
The dawn of gene-editing to cure inherited disease.

version 1 -- breaking sh*t

ir.crisprtx.com/news-releases/…
The diseases treated, sickle cell, beta-thalassemia, both caused wrongly shaped hemoglobin. that is what carries oxygen in the blood. painful diseases.
but some people with these diseases don't suffer much, because their bodies still produce fetal hemoglobin (whereas, in the rest of us, it's off).

One of many cases in which "strange" cases provide a map to treatment.
I think the way this treatment works is CRISPR employed to damage/disable expression of a protein called BCLA11, so less of that, which in turn allows expression of fetal hemoglobin again.

clever
These treatments seem to entail an autologous bone marrow transplant. Bone marrow cells removed, then some nasty chemo to kill off the rest. Then edited bone marrow cells returned to body, and hope it engrafts, or revives.
something not commented upon is that sibling bone marrow transplant already possible, but relatively little used. might indicate limited interest in this intensive treatment, esp. in resource poor places where diseases more common.
See this talk by Vence Bonham of the NIH on attitudes among sickle cell patients towards CRISPR
technologyreview.com/s/614360/crisp…

events.technologyreview.com/video/watch/ve…
Blog from NIH director describing the stratagem. directorsblog.nih.gov/2019/04/02/a-c…
Missing some Tweet in this thread? You can try to force a refresh.

Enjoying this thread?

Keep Current with Antonio Regalado

Profile picture

Stay in touch and get notified when new unrolls are available from this author!

Read all threads

This Thread may be Removed Anytime!

Twitter may remove this content at anytime, convert it as a PDF, save and print for later use!

Try unrolling a thread yourself!

how to unroll video

1) Follow Thread Reader App on Twitter so you can easily mention us!

2) Go to a Twitter thread (series of Tweets by the same owner) and mention us with a keyword "unroll" @threadreaderapp unroll

You can practice here first or read more on our help page!

Follow Us on Twitter!

Did Thread Reader help you today?

Support us! We are indie developers!


This site is made by just three indie developers on a laptop doing marketing, support and development! Read more about the story.

Become a Premium Member ($3.00/month or $30.00/year) and get exclusive features!

Become Premium

Too expensive? Make a small donation by buying us coffee ($5) or help with server cost ($10)

Donate via Paypal Become our Patreon

Thank you for your support!