CRISPR 临床试验的五年进展(2019-2024 年)。

IF 3.7 4区 生物学 Q2 GENETICS & HEREDITY CRISPR Journal Pub Date : 2024-10-01 DOI:10.1089/crispr.2024.0081
Kevin Davies, Alex Philippidis, Rodolphe Barrangou
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引用次数: 0

摘要

2019 年 7 月,维多利亚-格雷作为志愿者参加了由 Vertex 制药公司和 CRISPR Therapeutics 赞助的 exa-cel 临床试验,成为第一位接受基于 CRISPR 的细胞疗法的镰状细胞病患者。仅仅四年后,这种被命名为 Casgevy 的疗法就获得了欧洲、美国和中东监管机构的批准,开创了 CRISPR 药物的新时代。在此期间,数十项其他临床试验也相继启动,包括许多正在全球招募患者的一期、二期和三期临床试验。在这篇简短的《透视》中,我们整理了以CRISPR、基质和质粒编辑为特色的治疗性临床试验的最新信息,涉及一系列体内和体外基因和细胞疗法。
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Five Years of Progress in CRISPR Clinical Trials (2019-2024).

In July 2019, Victoria Gray became the first patient with sickle cell disease to receive a CRISPR-based cell therapy as a volunteer in the exa-cel clinical trial, sponsored by Vertex Pharmaceuticals and CRISPR Therapeutics. Barely four years later, the ensuing therapy, branded as Casgevy, received approval from regulatory agencies in Europe, the United States, and the Middle East, ushering in a new era of CRISPR-based medicines. During this period, scores of other clinical trials have been launched, including many actively recruiting patients across phase 1, phase 2, and phase 3 clinical trials around the world. In this brief Perspective, we collate the latest information on therapeutic clinical trials featuring CRISPR, base and prime editing, across a range of both in vivo and ex vivo gene and cell therapies.

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来源期刊
CRISPR Journal
CRISPR Journal Biochemistry, Genetics and Molecular Biology-Biotechnology
CiteScore
6.30
自引率
2.70%
发文量
76
期刊介绍: In recognition of this extraordinary scientific and technological era, Mary Ann Liebert, Inc., publishers recently announced the creation of The CRISPR Journal -- an international, multidisciplinary peer-reviewed journal publishing outstanding research on the myriad applications and underlying technology of CRISPR. Debuting in 2018, The CRISPR Journal will be published online and in print with flexible open access options, providing a high-profile venue for groundbreaking research, as well as lively and provocative commentary, analysis, and debate. The CRISPR Journal adds an exciting and dynamic component to the Mary Ann Liebert, Inc. portfolio, which includes GEN (Genetic Engineering & Biotechnology News) and more than 80 leading peer-reviewed journals.
期刊最新文献
Early Detection of Wildlife Disease Pathogens Using CRISPR-Cas System Methods. CRISPR-GRIT: Guide RNAs with Integrated Repair Templates Enable Precise Multiplexed Genome Editing in the Diploid Fungal Pathogen Candida albicans. Genome Editing in Apicomplexan Parasites: Current Status, Challenges, and Future Possibilities. Mutation-Specific CRISPR Targeting with SaCas9 and AsCas12a Restores Therapeutic Sensitivity in Treatment-Resistant Melanoma. Characterization of Research Support of Genome Editing Technologies and Transition to Clinical Trials.
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