Gates Package — page 831

5. Wang H, Richter M, Psatha N, Li C, Kim J, Liu J, et al. A Combined In Vivo HSC Transduction/Selection Approach Results in Efficient and Stable Gene Expression in Peripheral Blood Cells in Mice. Molecular therapy Methods & clinical development. 2018;8:52-64. 6. Neff T, Horn PA, Peterson LJ, Thomasson BM, Thompson J, Williams DA, et al. Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model. J Clin Invest. 2003;112(10):1581-8. 7. Beard BC, Trobridge GD, Ironside C, McCune JS, Adair JE, and Kiem HP. Efficient and stable MGMTmediated selection of long-term repopulating stem cells in nonhuman primates. J Clin Invest. 2010;120(7):2345-54. 8. Li C, Wang H, Gil S, Germond A, Fountain C, Baldessari A, et al. Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors. Mol Ther Methods Clin Dev. 2022;24:127-41. 9. Wang H, Georgakopoulou A, Li C, Liu Z, Gil S, Bashyam A, et al. Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements. JCI Insight. 2020;5(16). 10. Wang H, Georgakopoulou A, Psatha N, Li C, Capsali C, Samal HB, et al. In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia. J Clin Invest. 2019;129(2):598-615. 11. Li C, Course MM, McNeish IA, Drescher CW, Valdmanis PN, and Lieber A. Prophylactic In Vivo Hematopoietic Stem Cell Gene Therapy with an Immune Checkpoint Inhibitor Reverses Tumor Growth in Syngeneic Mouse Tumor Models. Cancer Res. 2020;80(3):549-60. 12. Li C, Georgakopoulou A, Mishra A, Gil S, Hawkins RD, Yannaki E, et al. In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal gamma-globin in beta-YAC mice. Blood Adv. 2021;5(4):1122-35. 13. Li C, Mishra AS, Gil S, Wang M, Georgakopoulou A, Papayannopoulou T, et al. Targeted Integration and High-Level Transgene Expression in AAVS1 Transgenic Mice after In Vivo HSC Transduction with HDAd5/35++ Vectors. Mol Ther. 2019;27(12):2195-212. 14. Li C, Wang H, Georgakopoulou A, Gil S, Yannaki E, and Lieber A. In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model. Mol Ther. 2021;29(2):822-37. 15. Wang H, Liu Z, Li C, Gil S, Papayannopoulou T, Doering CB, et al. High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells. Blood Adv. 2019;3(19):2883-94.