In this case, mRNA-LNPs were capable of going for hematopoietic stem cells (HSCs)… …in order to produce a “gene editor” within the cell:
In another experiment, the researchers stuffed their nanoparticles with an mRNA sequence that produces a gene editor when it enters the cell. The editor targets a mutation in hemoglobin causing sickle cell disease.
So Vertex expects to only treat about 1200 people over the age of 12 with SCA?
Why, for what we wasted in either Afghanistan or Ukraine ...