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基因编辑CRISPR-Cas9的治疗应用

Aditya Bharti¹, Joann Mudge²

¹Green Level High School, Cary, NC, United States.

Frontiers in genome editing

|January 1, 2026

概括

在首次获得FDA批准后,CRISPR-Cas9基因编辑正在为包括癌症和传染病在内的各种疾病进行临床试验. 本综述涵盖了截至2024年底的正在进行的CRISPR-Cas9临床试验.

科学领域:

生物技术是生物技术.
遗传学是一个遗传学.
分子生物学分子生物学

背景情况:

克里斯普尔-Cas9是一种革命性的基因编辑技术,广泛用于研究.
第一个CRISPR疗法于2023年底被FDA批准,标志着一个重要的里程碑.
在当前的基因编辑疗法临床试验中,CRISPR-Cas9是主要的工具.

研究的目的:

审查在clinicaltrials.gov.gov上注册的新兴CRISPR-Cas9临床试验.
突出了CRISPR-Cas9在最初的批准之外不断扩大的治疗应用.
提供CRISPR-Cas9在治疗各种疾病中的作用的概述.

主要方法:

在clinicaltrials.gov.gov注册的临床试验的系统审查.
数据收集集中在涉及CRISPR-Cas9技术的试验上.
截至2024年12月31日,对试验数据的分析.

主要成果:

一种基于CRISPR的疗法已被批准用于状细胞贫血和依赖输血的β-thalassemia.
临床试验正在探索CRISPR-Cas9的广泛目标,包括癌症和传染病.
注册的CRISPR-Cas9临床试验数量表明治疗兴趣日益增加.

结论:

克里斯普尔-Cas9技术正在迅速从实验室发展到临床应用.
针对CRISPR-Cas9疗法的疾病范围正在显著扩大.
目前正在进行的临床试验强调了CRISPR-Cas9基因编辑的治疗潜力.

关键词:

在CRISPR-cas9中使用.癌症癌症癌症癌症癌症临床试验是指临床试验中的临床试验.基因编辑基因编辑治疗药物治疗药物

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