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实现 CRISPR 基因疗法

IF 12.9 1区 生物学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY
Nature chemical biology Pub Date : 2024-06-13 DOI:10.1038/s41589-024-01645-x
Yitong Ma, Lei S. Qi
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引用次数: 0

摘要

首款基于 CRISPR 的药物 Casgevy 已获得多个医疗机构的批准，其他基于 CRISPR 的疗法目前也在临床试验中。虽然还有一些技术障碍需要克服，但化学生物学在将最近在碱基编辑、质粒编辑和表观遗传编辑方面取得的突破发展成未来的治疗方法方面发挥着至关重要的作用。
本文章由计算机程序翻译，如有差异，请以英文原文为准。

The realization of CRISPR gene therapy

The realization of CRISPR gene therapy

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The realization of CRISPR gene therapy
The inaugural CRISPR-based drug Casgevy has been approved by several medical agencies, with other CRISPR-based therapies currently in clinical trials. Although there are technological hurdles to overcome, chemical biology has a vital role in developing recent breakthroughs in base editing, prime editing and epigenetic editing into future treatments.
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来源期刊
Nature chemical biology
Nature chemical biology 生物-生化与分子生物学
CiteScore
23.90
自引率
1.40%
发文量
238
审稿时长
12 months
期刊介绍： Nature Chemical Biology stands as an esteemed international monthly journal, offering a prominent platform for the chemical biology community to showcase top-tier original research and commentary. Operating at the crossroads of chemistry, biology, and related disciplines, chemical biology utilizes scientific ideas and approaches to comprehend and manipulate biological systems with molecular precision. The journal embraces contributions from the growing community of chemical biologists, encompassing insights from chemists applying principles and tools to biological inquiries and biologists striving to comprehend and control molecular-level biological processes. We prioritize studies unveiling significant conceptual or practical advancements in areas where chemistry and biology intersect, emphasizing basic research, especially those reporting novel chemical or biological tools and offering profound molecular-level insights into underlying biological mechanisms. Nature Chemical Biology also welcomes manuscripts describing applied molecular studies at the chemistry-biology interface due to the broad utility of chemical biology approaches in manipulating or engineering biological systems. Irrespective of scientific focus, we actively seek submissions that creatively blend chemistry and biology, particularly those providing substantial conceptual or methodological breakthroughs with the potential to open innovative research avenues. The journal maintains a robust and impartial review process, emphasizing thorough chemical and biological characterization.
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