CRISPR-Cas12a Gene Editing of
 HBG1
 and
 HBG2
 Promoters to Treat β-Thalassemia

Haydar Frangoul; Rabi Hanna; Mark C. Walters; Roy L. Kao; Clinton Carroll; Meghann McManus; Kai-Hsin Chang; Michael C. Jaskolka; Keunpyo Kim; Qifeng Yu; Nnenna Badamosi; Baisong Mei; Olubunmi Afonja; Alexis Thompson

doi:10.1056/nejmoa2501277

CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-Thalassemia

H Haydar Frangoul (Sarah Cannon Research Institute at the Children’s Hospital at TriStar Centennial, Nashville) R Rabi Hanna (Department of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, Cleveland Clinic, Cleveland) M Mark C. Walters (UCSF Benioff Children’s Hospital, Oakland, CA) R Roy L. Kao (Division of Hematology, Oncology, and Transplantation, University of Minnesota, Minneapolis) C Clinton Carroll (Sarah Cannon Research Institute, Nashville) M Meghann McManus (Sarah Cannon Research Institute, Nashville) K Kai-Hsin Chang (Department of Chemistry) M Michael C. Jaskolka (Editas Medicine, Cambridge, MA) K Keunpyo Kim (Editas Medicine, Cambridge, MA) Q Qifeng Yu N Nnenna Badamosi (Editas Medicine, Cambridge, MA) B Baisong Mei (Editas Medicine, Cambridge, MA) O Olubunmi Afonja (Editas Medicine, Cambridge, MA) A Alexis Thompson

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Article Details

Journal New England Journal of Medicine

Volume / Issue Vol. 394, Issue 13

Published April 02, 2026

Pages 1292-1301

DOI 10.1056/nejmoa2501277

ISSN 0028-4793

Publisher Massachusetts Medical Society

Journal Info

New England Journal of Medicine

Massachusetts Medical Society

ISSN: 0028-4793 Social Sciences

Authors (14)

H

Haydar Frangoul

Sarah Cannon Research Institute at the Children’s Hospital at TriStar Centennial, Nashville

R

Rabi Hanna

Department of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, Cleveland Clinic, Cleveland

M

Mark C. Walters

UCSF Benioff Children’s Hospital, Oakland, CA

R

Roy L. Kao

Division of Hematology, Oncology, and Transplantation, University of Minnesota, Minneapolis

C

Clinton Carroll

Sarah Cannon Research Institute, Nashville

M

Meghann McManus

Sarah Cannon Research Institute, Nashville

K

Kai-Hsin Chang

Department of Chemistry

M

Michael C. Jaskolka