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New England Journal of Medicine2021Frangoul et al.The first published clinical results showing CRISPR editing produced benefit in patients.
Research paper · Clinical
CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia
The first published clinical results showing CRISPR editing produced benefit in patients.
Simple explanation
This paper reported the first two patients, one with sickle cell disease and 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.
Go deeper
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.
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