CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

Frangoul, Haydar; Altshuler, David; Domenica Cappellini, Maria; Chen, Yi-Shan; Domm, Jennifer; K. Eustace, Brenda; Foell, Juergen; de la Fuente, Josu; A. Grupp, Stephan; Handgretinger, Rupert; W. Ho, Tony; Kattamis, Antonis; Kernytsky, Andrew; Lekstrom-Himes, Julie; M. Li, Amanda; Locatelli, Franco; Y. Mapara, Markus; de Montalembert, Mariane; Rondelli, Damiano; Sharma, Akshay; Sheth, Sujit; Soni, Sandeep; H. Steinberg, Martin; A. Wall, Donna; Yen, Angela; Corbacioglu, Selim

doi:10.1056/nejmoa2031054

Public

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

Published in New England Journal of Medicine • Dec 5, 2020

Authors:

Haydar Frangoul

David Altshuler

Maria Domenica Cappellini

Abstract

Transfusion-dependent -thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is a transcription factor that represses -globin expression and fetal hemoglobin in erythroid cells. We performed electroporation o...

Subject

CRISPR

Thalassemia

Genome editing

Research Assistant

AI chat, annotations, notes & similar papers

Finding related papers...

Discussions

(0)

No comments yet

Be the first to share your thoughts!