相关参考文献
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Prime editing efficiency and fidelity are enhanced in the absence of mismatch repair
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Intracellular RNase activity dampens zinc finger nuclease-mediated gene editing in hematopoietic stem and progenitor cells
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High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation
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Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells
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Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
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Base editing of haematopoietic stem cells rescues sickle cell disease in mice
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Modeling a cataract disorder in mice with prime editing
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Targeted mutagenesis in mouse cells and embryos using an enhanced prime editor
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Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression
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Cas9 activates the p53 pathway and selects for p53-inactivating mutations
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Efficient generation of mouse models with the prime editing system
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Therapeutic base editing of human hematopoietic stem cells
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Evidence for ineffective erythropoiesis in severe sickle cell disease
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Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia
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