CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease

Author:

Sharma Akshay1ORCID,Boelens Jaap-Jan1ORCID,Cancio Maria1,Hankins Jane S.1,Bhad Prafulla1,Azizy Marjohn1,Lewandowski Andrew1,Zhao Xiaojun1,Chitnis Shripad1,Peddinti Radhika1,Zheng Yan1,Kapoor Neena1,Ciceri Fabio1,Maclachlan Timothy1,Yang Yi1,Liu Yi1,Yuan Jianping1,Naumann Ulrike1,Yu Vionnie W.C.1,Stevenson Susan C.1,De Vita Serena1,LaBelle James L.1

Affiliation:

1. From St. Jude Children’s Research Hospital, Memphis, TN (A.S., J.S.H., Y.Z.); Memorial Sloan Kettering Cancer Center, New York (J.-J.B., M.C.); the Novartis Institutes for BioMedical Research, Cambridge, MA (P.B., M.A., A.L., X.Z., S.C., T.M., Y.Y., Y.L., J.Y., V.W.C.Y., S.C.S., S.D.V.); University of Chicago Medicine, Chicago (R.P., J.L.L.); Children’s Hospital Los Angeles, Los Angeles (N.K.); IRCCS San Raffaele Hospital, Milan (F.C.); and the Novartis Institutes for BioMedical Research, Basel,...

Funder

Novartis Pharmaceuticals Corporation

Publisher

Massachusetts Medical Society

Subject

General Medicine

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