Prime time for gene editing

IF 58.7 1区 医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY Nature Medicine Pub Date : 2024-12-13 DOI:10.1038/s41591-024-03390-z
Karen O’Leary
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基因编辑的黄金时期
基因编辑工具发展迅速,从最初的 CRISPR-Cas 系统到碱基编辑,再到最近的质粒编辑。与 CRISPR-Cas 系统不同,碱基编辑不需要双链断裂;质粒编辑也不需要双链断裂,而且它支持更强的编辑能力,包括定向插入和删除。今年 4 月,美国食品和药物管理局批准了 PM359(Prime Medicine 公司生产)的新药研究申请,使其成为第一个进入临床的素编辑器。PM359被设计为纠正导致慢性肉芽肿病的突变的一次性疗法,将首先在一项1/2期研究(NCT06559176)中对成年患者进行评估,如果认为安全,还将在青少年和儿童中进行测试。另一种技术是表观基因组编辑,它能在不改变基因本身的情况下实现基因沉默。相反,编辑机制针对的是基因周围决定基因表达的可遗传(但可逆)结构。虽然这种技术仍处于临床前阶段,但它为一系列新的基因编辑工具增添了新的内容,有望带来更大的灵活性和安全性。原始参考文献:《首要医学》https://go.nature.com/4esxKzk(2024 年 4 月 29 日);《自然》627, 416-423 (2024)
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来源期刊
Nature Medicine
Nature Medicine 医学-生化与分子生物学
CiteScore
100.90
自引率
0.70%
发文量
525
审稿时长
1 months
期刊介绍: Nature Medicine is a monthly journal publishing original peer-reviewed research in all areas of medicine. The publication focuses on originality, timeliness, interdisciplinary interest, and the impact on improving human health. In addition to research articles, Nature Medicine also publishes commissioned content such as News, Reviews, and Perspectives. This content aims to provide context for the latest advances in translational and clinical research, reaching a wide audience of M.D. and Ph.D. readers. All editorial decisions for the journal are made by a team of full-time professional editors. Nature Medicine consider all types of clinical research, including: -Case-reports and small case series -Clinical trials, whether phase 1, 2, 3 or 4 -Observational studies -Meta-analyses -Biomarker studies -Public and global health studies Nature Medicine is also committed to facilitating communication between translational and clinical researchers. As such, we consider “hybrid” studies with preclinical and translational findings reported alongside data from clinical studies.
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