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Letters to the Editor
cells of beta-thalassemia and sickle cells disease patients. PLoS One.
2012;7(3):32345. 9.GudaS,BrendelC,RenellaR,etal.,miRNA-embeddedshRNAsfor
lineage-specific BCL11A knockdown and hemoglobin F induction.
Mol Ther. 2015;23(9):1465-1474.
10. Brendel C, Guda S, Renella R, et al. Lineage-specific BCL11A knock-
down circumvents toxicities and reverses sickle phenotype. J Clin
Invest. 2016;126(10):3868-3878.
11. Fellmann C, Hoffmann T, Sridhar V, et al., An optimized microRNA
backbone for effective single-copy RNAi. Cell Rep. 2013;5(6):1704-
1713.
12. Kurita R, Suda N, Sudo K, et al., Establishment of immortalized
human erythroid progenitor cell lines able to produce enucleated red
blood cells. PLoS One. 2013;8(3):59890.
13.Luc S, Huang J, McEldoon JL, et al. Bcl11a-deficiency leads to
hematopoietic stem cell defects with an aging-like phenotype. Cell
Rep. 2016;16(12):3181-3194.
14. Psatha N, Reik A, Phelps S, et al. Disruption of the BCL11A erythroid
enhancer reactivates fetal hemoglobin in erythroid cells of patients with b-thalassemia major. Mol Ther Methods Clin Dev. 2018; 10:313-326.
15. Breda L, Ghiaccio V, Tanaka N, et. al. Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies. Mol Ther. 2021;29(4):1625-1638.
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