Gates Package — page 815

In vivo base editing by a single intravenous vector injection for treatment of hemoglobinopathies Chang Li1, Aphrodite Georgakopoulou2, Gregory A. Newby3, 4, 5, Kelcee A. Everette3, 4, 5, Evangelos Nizamis6, Kiriaki Paschoudi 2,7, Efthymia Vlachaki8, Sucheol Gil1, Anna K. Anderson1, Theodore Koob1, Lishan Huang1, Hongjie Wang1, Hans-Peter Kiem9, David R. Liu3, 4, 5, Evangelia Yannaki2, André Lieber1,10 1University of Washington, Department of Medicine, Division of Medical Genetics, Seattle, WA 98195 2Gene and Cell Therapy Center, Hematology Department, George Papanicolaou Hospital, Thessaloniki, Greece 3Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA 4Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA 5Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA 6University of Thessaly, Department of Computer Science and Biomedical Informatics, Lamia, Greece 7Aristotle University of Thessaloniki, School of Biology, Thessaloniki, Greece 8Hematological Laboratory, Second Department of Internal Medicine, Faculty of Health Sciences, School of Medicine, Aristotle University of Thessaloniki, Hippokration General Hospital, Thessaloniki, Greece 9Stem and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109 10University of Washington, Department of Pathology, Seattle, WA 98195 Conflict of interest: HPK is a paid advisor for Ensoma Bio. Corresponding authors: André Lieber: University of Washington, Box 357720, Seattle, WA 98195, phone: email: @uw.edu, phone: Chang Li: University of Washington, Box 357720, Seattle, WA 98195, phone: email: @uw.edu