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

Author:

Frangoul Haydar1,Altshuler David1,Cappellini M. Domenica1,Chen Yi-Shan1,Domm Jennifer1,Eustace Brenda K.1,Foell Juergen1,de la Fuente Josu1,Grupp Stephan1,Handgretinger Rupert1,Ho Tony W.1,Kattamis Antonis1,Kernytsky Andrew1,Lekstrom-Himes Julie1,Li Amanda M.1,Locatelli Franco1,Mapara Markus Y.1,de Montalembert Mariane1,Rondelli Damiano1,Sharma Akshay1,Sheth Sujit1,Soni Sandeep1,Steinberg Martin H.1,Wall Donna1,Yen Angela1,Corbacioglu Selim1

Affiliation:

1. From the Sarah Cannon Center for Blood Cancer at the Children’s Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children’s Research Hospital, Memphis (A.S.) — both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) — both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza,...

Funder

CRISPR Therapeutics AG

Vertex Pharmaceuticals Incorporated

Publisher

Massachusetts Medical Society

Subject

General Medicine

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