Synthetic Nanobiotechnology and Biomachines, Synthetic Biology and Precision Medicine Centre, Future production Chemicals Cluster, Council for Scientific and Industrial Research, Pretoria, South Africa.
Synthetic Nanobiotechnology and Biomachines, Synthetic Biology and Precision Medicine Centre, Future production Chemicals Cluster, Council for Scientific and Industrial Research, Pretoria, South Africa; Department of Surgery, University of the Witwatersrand, Johannesburg, South Africa.
Prog Mol Biol Transl Sci. 2024;208:261-284. doi: 10.1016/bs.pmbts.2024.07.004. Epub 2024 Aug 26.
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.
CRISPR-Cas 系统通过使个性化治疗针对个体的基因谱,彻底改变了精准医学。已经开发了各种 CRISPR 技术来靶向血液癌症中的特定致病基因,并且一些技术已经进入临床试验。尽管一些研究已经探索了 CRISPR-Cas 系统的体内应用,但仍有几个挑战继续阻碍其广泛应用。此外,CRISPR-Cas 技术在提高免疫疗法对血液癌症的反应方面显示出很大的潜力。CAR-T 细胞疗法的出现已经在血液癌症中靶向和纠正致病基因方面取得了相当大的成功。尽管 CRISPR-Cas 在治疗血液癌症方面具有巨大的潜力,但与安全性、伦理和监管批准相关的问题仍然是重大障碍。本综述强调了 CRISPR-Cas 技术在彻底改变血液癌症治疗方面的变革潜力。