3 ms·
Could we do the same thing with sickle cell?
by mrfusion 9y ago
Could we do the same thing with sickle cell?
- Nomentatus 9y agoSince that trait can be viewed as the double-absence of the statistically "normal" gene, I would guess yes. The near normal protein levels in this trial (despite the fact that only a small percentage of cells were likely altered) are a very positive sign.
- refurb 9y agoYes, there is a company whose name escapes me working on it - Bluebird? You insert the correct hemoglobin gene and express it at a high enough level that you drive the percentage of sickle hemoglobin low enough that you no longer get the sickle cell crises. Last I read they were having trouble getting durable expression.