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CRISPR-Mediated Base Editing: Promises and Challenges for a Viable Oncotherapy Strategy.

Human gene therapy(2023)

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摘要
Base editing technology, developed from the CRISPR/Cas9 system, is able to efficiently implement single base substitutions at specific DNA or RNA sites without generating double-strand breaks (DSBs) with precision and efficiency. Point mutations account for 58% of disease-causing genetic mutations in humans, and single nucleotide variants (SNVs) are an important cause of tumorigenesis, and the advent of base editors offers new hope for the study or treatment of such diseases. Although it has some limitations, base editors have been continuously improved in terms of editing efficiency, specificity and product purity since their development. In this review, we examine the main base editing technologies and discuss their applications and prospects in tumor research and therapy, as well as elaborate on their mode of delivery.
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关键词
base editing, SNVs, delivery method, tumor, gene therapy
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