3 ms·
The mechanism by which this treatment works is really neat (some notes in [0]). The treatment increases the production of a fetal version of hemoglobin. That fe
by EvanAnderson 3y ago
The mechanism by which this treatment works is really neat (some notes in [0]). The treatment increases the production of a fetal version of hemoglobin. That fetal hemoglobin is unaffected by the sickle cell mutation. Presumably everybody who is alive (and not a fetus) has a good copy of this fetal hemoglobin gene and it "just" needs re-activated.
[0] https://sciencebasedmedicine.org/first-crispr-treatment-approval/ https://sciencebasedmedicine.org/first-crispr-treatment-appr...