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Article

Therapeutic adenine base editor with minimized off-target effects.

Protein & cell - 1 Jun 2026

Sun Yongsen, Yan Nana, Feng Hu, Lu Hongjiang, Zuo Zhenrui, Zhou Chikai, Zuo Erwei

Abstract excerpt

Genome-wide off-target effect poses a safety risk for clinical use of adenine base editor (ABE), among which ABE8e is one of the most efficient. Genome-wide off-target analysis by two-cell embryo injection (GOTI) analysis showed that the rate of genome-wide single-nucleotide variants (SNVs) in ABE8e-edited cells was ∼30-fold higher than that of spontaneous SNVs in control cells, indicating prevalent off-target...

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Adenine
Aminohydrolases
CRISPR-Cas Systems
PAM gene
Genetic Therapy
Mice

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Polymorphism, Single Nucleotide

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Identifiers

PMID: 41645451
DOI: 10.1093/procel/pwag006
PMCID: PMC13225742

Genes

PAM

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 1 Sept 2026.

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