[robwwilliams]

Good point and thanks for being on-topic. Humans have three variants of the HBB gene and having sickling mutations in the variant expressed in adult is causal to SC disease.

The FDA-approved treatments reactivate the fetal HBB gene in adults and this change in gene expression control effectively prevents SCD.

Very cool and transformative work. Now we have to get the price tag down from seven figures to four or five figures so that it will be used widely. That may be a few decades. Let’s hope that more efficient alternatives are developed soon.

[firejake308]

From an efficiency standpoint, I think having to harvest and modify the patient's stem cells is probably the biggest choke point, right? I would imagine that if you could inject something once and be done with it (I'm thinking like Zolgensma), you could mass produce it more effectively