A therapy that uses CRISPR-Cas9 to edit the blood stem cells of people with sickle cell disease before reinfusing the cells was safe and effective, according to results from a phase 1 and 2 clinical study involving 3 adult participants with the condition. By increasing the amount of fetal hemoglobin in participants’ red blood cells, the treatment protected against some of the effects of sickle cell disease, a condition that affects about 100 000 people in the US.
Source: JAMA Online First