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Base editing of key residues in the BCL11A-XL-specific zinc finger domains derepresses fetal globin expression.
Rajendiran V, Devaraju N, Haddad M, Ravi NS, Panigrahi L, Paul J, Gopalakrishnan C, Wyman S, Ariudainambi K, Mahalingam G, Periyasami Y, Prasad K, George A, Sukumaran D, Gopinathan S, Pai AA, Nakamura Y, Balasubramanian P, Ramalingam R, Thangavel S, Velayudhan SR, Corn JE, Mackay JP, Marepally S, Srivastava A, Crossley M, Mohankumar KM. Rajendiran V, et al. Mol Ther. 2024 Mar 6;32(3):663-677. doi: 10.1016/j.ymthe.2024.01.023. Epub 2024 Jan 24. Mol Ther. 2024. PMID: 38273654 Free article.