간단한 설명
This paper reported the first two patients — one with sickle cell disease, one with beta thalassemia — treated with the editing therapy that later became Casgevy. Both had large increases in fetal haemoglobin and stopped having the problems that define their diseases.
더 깊이 알아보기
Frangoul and colleagues reported results in the first two patients treated with CTX001, showing high levels of fetal haemoglobin, transfusion independence in the thalassemia patient and absence of vaso-occlusive crises in the sickle cell patient, with a safety profile consistent with myeloablative conditioning.
Sources
- New England Journal of Medicine · 2021
CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia ↗