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CRISPR-Cas 系统在血液肿瘤中的进展

英文原题:Advances in CRISPR-Cas systems for blood cancer.

查看英文原题

Advances in CRISPR-Cas systems for blood cancer.

PubMed 2024/08/26(内容时间) Prog Mol Biol Transl Sci

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中文摘要

CRISPR-Cas系统通过实现针对个体遗传特征的个性化治疗,彻底改变了精准医学。多种CRISPR技术已被开发用于靶向血液肿瘤中的特定致病基因,其中一些已进入临床试验。尽管一些研究探索了CRISPR-Cas系统的体内应用,但若干挑战仍阻碍其广泛应用。此外,CRISPR-Cas技术在改善免疫疗法对血液肿瘤的应答方面已显示出前景。CAR-T 细胞疗法的出现已在靶向和纠正血液肿瘤致病基因方面取得显著成功。尽管CRISPR-Cas在血液肿瘤治疗中具有广阔前景,但安全性、伦理和监管审批相关问题仍是重大障碍。这篇综合综述强调了CRISPR-Cas技术彻底变革血液肿瘤治疗的转化潜力。

展开英文摘要原文

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.

论文信息

作者
Monchusi B、Dube P、Takundwa MM、Kenmogne VL、Thimiri Govinda Raj DB
第一作者单位
Synthetic Nanobiotechnology and Biomachines, Synthetic Biology and Precision Medicine Centre, Future production Chemicals Cluster, Council for Scientific and Industrial Research, Pretoria, South Africa.
通讯作者单位
Synthetic Nanobiotechnology and Biomachines, Synthetic Biology and Precision Medicine Centre, Future production Chemicals Cluster, Council for Scientific and Industrial Research, Pretoria, South Africa. Electronic address: dgovindaraj@csir.co.za.
文献类型
综述
期刊
Progress in molecular biology and translational science2024
原文标识
PubMed 39266186 · DOI 10.1016/bs.pmbts.2024.07.004