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30. Richter MF, Zhao KT, Eton E, Lapinaite A, Newby GA, Thuronyi BW, et al. Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity. Nat Biotechnol. 2020;38(7):883-91. 31. Martyn GE, Wienert B, Kurita R, Nakamura Y, Quinlan KGR, and Crossley M. A natural regulatory mutation in the proximal promoter elevates fetal globin expression by creating a de novo GATA1 site. Blood. 2019;133(8):852-6. 32. Saydaminova K, Ye X, Wang H, Richter M, Ho M, Chen H, et al. Efficient genome editing in hematopoietic stem cells with helper-dependent Ad5/35 vectors expressing site-specific endonucleases under microRNA regulation. Mol Ther Methods Clin Dev. 2015;1:14057. 33. Palmer DJ, Turner DL, and Ng P. Production of CRISPR/Cas9-Mediated Self-Cleaving HelperDependent Adenoviruses. Mol Ther Methods Clin Dev. 2019;13:432-9. 34. Li C, Psatha N, Gil S, Wang H, Papayannopoulou T, and Lieber A. HDAd5/35(++) Adenovirus Vector Expressing Anti-CRISPR Peptides Decreases CRISPR/Cas9 Toxicity in Human Hematopoietic Stem Cells. Mol Ther Methods Clin Dev. 2018;9:390-401. 35. Sripichai O, Kiefer CM, Bhanu NV, Tanno T, Noh SJ, Goh SH, et al. Cytokine-mediated increases in fetal hemoglobin are associated with globin gene histone modification and transcription factor reprogramming. Blood. 2009;114(11):2299-306. 36. Journal of Gene Medicine website, www.wiley.co.uk/genmed/clinical/. 37. Li C, Psatha N, Sova P, Gil S, Wang H, Kim J, et al. Reactivation of gamma-globin in adult beta-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing. Blood. 2018;131(26):291528. 38. Cheng Y, and Tsai SQ. Illuminating the genome-wide activity of genome editors for safe and effective therapeutics. Genome Biol. 2018;19(1):226. 39. Gomez-Auli A, Hillebrand LE, Christen D, Gunther SC, Biniossek ML, Peters C, et al. The secreted inhibitor of invasive cell growth CREG1 is negatively regulated by cathepsin proteases. Cell Mol Life Sci. 2021;78(2):733-55. 40. Eaton WA, and Bunn HF. Treating sickle cell disease by targeting HbS polymerization. Blood. 2017;129(20):2719-26. 41. Piel FB, Steinberg MH, and Rees DC. Sickle Cell Disease. N Engl J Med. 2017;376(16):1561-73. 42. Steinberg MH. Fetal hemoglobin in sickle cell anemia. Blood. 2020;136(21):2392-400. 43. Persons DA, Hargrove PW, Allay ER, Hanawa H, and Nienhuis AW. The degree of phenotypic correction of murine beta -thalassemia intermedia following lentiviral-mediated transfer of a

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