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, University of Rome, Rome (F.L.); the University of Regensburg, Regensburg (J. Foell, S.C.), and Children's University Hospital, University of Tübingen, Tübingen (R.H.) — both in Germany; Imperial College Healthcare NHS Trust, St. Mary's Hospital, London (J. de la Fuente); Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.G.); the University of Athens, Athens (A. Kattamis); BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.), and the Hospital for Sick Children–University of Toronto, Toronto (D.W.) — both in Canada; Columbia University (M.Y.M.) and the Joan and Sanford I. Weill Medical College of Cornell University (S. Sheth), New York; Necker–Enfants Malades Hospital, Assistance Publique–Hôpitaux de Paris, University of Paris, Paris (M.M.); and the University of Illinois at Chicago, Chicago (D.R.).
Address reprint requests to Dr. Frangoul at the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, 330 23rd Ave. N., Suite 450, Nashville, TN 37203, or at [email protected]; or to Dr. Corbacioglu at Children's Hospital Regensburg, University of Regensburg, Franz-Josef Strauss Allee 11, 93053 Regensburg, Germany, or at [email protected].
Disclosure forms provided by the authors are available with the full text of this article at NEJM.org.
This article was published on December 5, 2020, and updated on December 7, 2020, at NEJM.org.
A data sharing statement provided by the authors is available with the full text of this article at NEJM.org.
We thank the patients and their families for their participation in these studies; the staff at our study sites for their contributions to the care of the patients; John Wu of BC Children's Hospital and Jane Hankins of St. Jude Children's Research Hospital; Christine Fernandez, Delphine Ranslant, Jennie Webb, Yimeng Lu, Jamie Doyle, Niraj Shanbhag, Sarah Mulcahey, Sorana Ailinca-Luchian, Lisa Moritz, Yael Bobruff, Suzan Imren, Jason Barber, Michael Weinstein, Gary Rea, John Chapin, Ewalina Morawa, Duyen Ngo, Sara Spencer, Parin Sripakdeevong, and Charis Campbell for their contributions to the conduct of these studies; Leah Eardley and Nathan Blow for their editorial support in the preparation of the manuscript; and Complete HealthVizion in Chicago for providing editorial assistance.
