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在CRISPR临床试验中的五年进展 (2019-2024)

Kevin Davies¹, Alex Philippidis², Rodolphe Barrangou^1,3

¹The CRISPR Journal, Mary Ann Liebert Inc., New Rochelle, New York, USA.

The CRISPR journal

|October 22, 2024

概括

克里斯普尔基因编辑正在彻底改变医学,首个基于克里斯普尔的状细胞疾病疗法在欧洲和美国获得批准. 这标志着基因和细胞疗法的新时代,世界各地正在进行众多临床试验.

科学领域:

生物技术是生物技术.
遗传学遗传学是一个
医学科学医学科学医学科学

背景情况:

首个基于CRISPR的状细胞疾病细胞疗法于2019年7月进行.
这种疗法,Casgevy,已经获得了欧洲,美国和中东的监管批准.
这一进步意味着基于CRISPR的药物进入了一个新时代.

研究的目的:

提供利用CRISPR技术进行治疗临床试验的现状概述.
突出基因和细胞疗法的进步,包括基因和原始编辑.
收集有关正在进行的临床试验的信息,包括体内和体外的方法.

主要方法:

审查最近的临床试验数据和监管批准.
汇编了各个阶段 (1,2,3) 的活跃临床试验信息.
专注于基因编辑技术,如CRISPR,基因编辑和原始编辑.

主要成果:

卡斯格维的成功开发和批准是一个重要的里程碑.
许多涉及基于CRISPR的疗法的临床试验正在全球积极招募患者.
使用体内和体外方法的广泛的基因和细胞疗法正在研究中.

结论:

克里斯普技术从最初的患者治疗到监管部门的批准和广泛的临床试验都迅速发展.
该领域正在扩大,包括基础和主要编辑,以及CRISPR.
目前正在进行的临床试验表明,基因和细胞疗法在治疗各种疾病方面具有前景.

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