治疗性基因编辑技术的最新进展。

IF 12.1 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
Dongqi Liu, Di Cao, Renzhi Han
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引用次数: 0

摘要

基因编辑技术的出现,特别是基于crispr的系统,已经彻底改变了生物医学研究和基因治疗的前景。正在进行的基因编辑研究导致了CRISPR技术的快速迭代,例如碱基和引物编辑器,可以在不产生有害的双链断裂(dsb)的情况下实现精确的核苷酸变化。此外,诸如DNA重组酶、DNA聚合酶和DNA连接酶的CRISPR融合系统的创新扩大了编辑序列的大小限制,为治疗开发开辟了新的途径。除了CRISPR系统之外,移动遗传元件(MGEs)和表观遗传编辑器正在成为哺乳动物细胞中精确大插入或稳定基因操作的有效替代方案。这些进步共同为下一代基因治疗的发展奠定了基础。这篇综述强调了遗传和表观遗传编辑工具的最新发展,并探讨了准备推进该领域的临床前创新。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Recent advances in therapeutic gene-editing technologies.

The advent of gene-editing technologies, particularly CRISPR-based systems, has revolutionized the landscape of biomedical research and gene therapy. Ongoing research in gene editing has led to the rapid iteration of CRISPR technologies, such as base and prime editors, enabling precise nucleotide changes without the need for generating harmful double-strand breaks (DSBs). Furthermore, innovations such as CRISPR fusion systems with DNA recombinases, DNA polymerases, and DNA ligases have expanded the size limitations for edited sequences, opening new avenues for therapeutic development. Beyond the CRISPR system, mobile genetic elements (MGEs) and epigenetic editors are emerging as efficient alternatives for precise large insertions or stable gene manipulation in mammalian cells. These advances collectively set the stage for next-generation gene therapy development. This review highlights recent developments of genetic and epigenetic editing tools and explores preclinical innovations poised to advance the field.

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来源期刊
Molecular Therapy
Molecular Therapy 医学-生物工程与应用微生物
CiteScore
19.20
自引率
3.20%
发文量
357
审稿时长
3 months
期刊介绍: Molecular Therapy is the leading journal for research in gene transfer, vector development, stem cell manipulation, and therapeutic interventions. It covers a broad spectrum of topics including genetic and acquired disease correction, vaccine development, pre-clinical validation, safety/efficacy studies, and clinical trials. With a focus on advancing genetics, medicine, and biotechnology, Molecular Therapy publishes peer-reviewed research, reviews, and commentaries to showcase the latest advancements in the field. With an impressive impact factor of 12.4 in 2022, it continues to attract top-tier contributions.
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