AZD7648 (DNA-PKcs inhibitor)：基因组编辑的双刃剑

IF 3.9 4区生物学 Q1 GENETICS & HEREDITY

Functional & Integrative Genomics Pub Date : 2025-03-01 DOI:10.1007/s10142-025-01560-x

Muhammad Waseem Sajjad, Ifrah Imran, Fatima Muzamil, Rubab Zahra Naqvi, Imran Amin

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引用次数: 0

摘要

聚类规则间隔短回文重复序列（CRISPR-Cas9）是过去十年中基因组编辑中最实用的技术。它的分子机制包括一系列步骤，从双链断裂到修复。在DNA链双链断裂后，通过同源定向修复（HDR）完成的DNA修复在不同的生物体中被认为是重要的，因为它是精确基因组编辑和减少意外突变的理想选择。然而，它主要由非同源末端连接（NHEJ）途径主导。Cullot等人最近发表在Nature Biotechnology上的一项研究显示AZD7648（一种DNA- pkcs抑制剂）在DNA修复时增加HDR事件的概率（Cullot et al. 2024）。

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AZD7648 (DNA-PKcs inhibitor): a two-edged sword for editing genomes

Clustered regularly interspaced short palindromic repeats (CRISPR-Cas9) has been the most practical technique in genome editing for the last decade. Its molecular mechanism includes steps that occur in a sequence, starting from a break in a double strand to repair. After a double-strand break in the DNA strand, the repairing of DNA done via Homology-Directed Repair (HDR) is considered important in different organisms as it is ideal for precise genome editing and the reduction of unintended mutations. Still, it is mostly dominated by the Non-Homologous End Joining (NHEJ) pathway. A recent study by Cullot et al. published in Nature Biotechnology showed interesting features of AZD7648 (a DNA-PKcs inhibitor) that increase the probability of HDR event while DNA repairing (Cullot et al. 2024).

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来源期刊

Functional & Integrative Genomics 生物-遗传学

CiteScore

3.50

自引率

3.40%

发文量

审稿时长

2 months

期刊介绍： Functional & Integrative Genomics is devoted to large-scale studies of genomes and their functions, including systems analyses of biological processes. The journal will provide the research community an integrated platform where researchers can share, review and discuss their findings on important biological questions that will ultimately enable us to answer the fundamental question: How do genomes work?