Advances in CRISPR-Cas systems for blood cancer.

3区生物学 Q2 Biochemistry, Genetics and Molecular Biology Progress in molecular biology and translational science Pub Date : 2024-01-01 Epub Date: 2024-08-26 DOI:10.1016/bs.pmbts.2024.07.004

Bernice Monchusi, Phumuzile Dube, Mutsa Monica Takundwa, Vanelle Larissa Kenmogne, Deepak Balaji Thimiri Govinda Raj

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Abstract

CRISPR-Cas systems have revolutionised precision medicine by enabling personalised treatments tailored to an individual's genetic profile. Various CRISPR technologies have been developed to target specific disease-causing genes in blood cancers, and some have advanced to clinical trials. Although some studies have explored the in vivo applications of CRISPR-Cas systems, several challenges continue to impede their widespread use. Furthermore, CRISPR-Cas technology has shown promise in improving the response of immunotherapies to blood cancers. The emergence of CAR-T cell therapy has shown considerable success in the targeting and correcting of disease-causing genes in blood cancers. Despite the promising potential of CRISPR-Cas in the treatment of blood cancers, issues related to safety, ethics, and regulatory approval remain significant hurdles. This comprehensive review highlights the transformative potential of CRISPR-Cas technology to revolutionise blood cancer therapy.

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CRISPR-Cas 系统在治疗血癌方面的进展。

CRISPR-Cas系统实现了针对个人基因特征的个性化治疗，从而彻底改变了精准医疗。针对血癌中的特定致病基因开发了各种 CRISPR 技术，其中一些已进入临床试验阶段。尽管一些研究已经探索了 CRISPR-Cas 系统在体内的应用，但仍有一些挑战阻碍了其广泛应用。此外，CRISPR-Cas 技术在改善免疫疗法对血癌的反应方面已显示出前景。CAR-T 细胞疗法的出现在靶向和纠正血癌致病基因方面取得了相当大的成功。尽管CRISPR-Cas在治疗血癌方面潜力巨大，但与安全性、伦理和监管审批相关的问题仍是重大障碍。本综述强调了 CRISPR-Cas 技术在彻底改变血癌治疗方面的变革潜力。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Progress in molecular biology and translational science 生物-生化与分子生物学

CiteScore

6.90

自引率

0.00%

发文量

审稿时长

>12 weeks

期刊介绍： Progress in Molecular Biology and Translational Science (PMBTS) provides in-depth reviews on topics of exceptional scientiﬁc importance. If today you read an Article or Letter in　Nature　or a Research Article or Report in　Science　reporting ﬁndings of exceptional importance, you likely will ﬁnd comprehensive coverage of that research area in a future　PMBTS　volume.

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