AsCas12a Gene Editing of HBG1/2 Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia

Hanna, R., Frangoul, H., McKinney, C., Pineiro, L., Mapara, M., Dalal, J., Chang, K.-H., Jaskolka, M., Kim, K., Farrington, D. L., Wally, M., Mei, B., Lawal, A., Afonja, O. O., & Walters, M. C. (2023). AsCas12a Gene Editing of HBG1/2 Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia. Blood, 142(Supplement 1), 4996–4996. https://doi.org/10.1182/blood-2023-187397

Authors:

Rabi Hanna

Haydar Frangoul

Christopher McKinney

Luis Piñeiro

Markus Y. Mapara

Jignesh Dalal

Kai‐Hsin Chang

Michael C. Jaskolka

Keunpyo Kim

Daphne L. Farrington

Meghan K. Wally