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ABE8e-NG is a high-activity CRISPR-based adenine base editor (ABE) designed to convert adenine (A) to guanine (G) in DNA without inducing double-strand breaks. It utilizes an evolved TadA deaminase domain (ABE8e), which features eight mutations that significantly enhance catalytic activity compared to earlier versions like ABE7.10. The "NG" variant incorporates a modified SpCas9 protein (SpCas9-NG) that recognizes an NGN protospacer adjacent motif (PAM), thereby expanding the genomic targeting range beyond the traditional NGG PAM. This tool has been investigated for the treatment of various genetic disorders, including TGFBI-linked corneal dystrophies, recessive dystrophic epidermolysis bullosa (RDEB), and retinitis pigmentosa, as well as for multiplex editing in hematopoietic stem cells to reactivate fetal hemoglobin.
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