Corporate Research Material Labs, 3M Center, 3M Company, Maplewood, MN 55144, USA.
Cells. 2023 Apr 7;12(8):1103. doi: 10.3390/cells12081103.
CRISPR-Cas technology has rapidly changed life science research and human medicine. The ability to add, remove, or edit human DNA sequences has transformative potential for treating congenital and acquired human diseases. The timely maturation of the cell and gene therapy ecosystem and its seamless integration with CRISPR-Cas technologies has enabled the development of therapies that could potentially cure not only monogenic diseases such as sickle cell anemia and muscular dystrophy, but also complex heterogenous diseases such as cancer and diabetes. Here, we review the current landscape of clinical trials involving the use of various CRISPR-Cas systems as therapeutics for human diseases, discuss challenges, and explore new CRISPR-Cas-based tools such as base editing, prime editing, CRISPR-based transcriptional regulation, CRISPR-based epigenome editing, and RNA editing, each promising new functionality and broadening therapeutic potential. Finally, we discuss how the CRISPR-Cas system is being used to understand the biology of human diseases through the generation of large animal disease models used for preclinical testing of emerging therapeutics.
CRISPR-Cas 技术迅速改变了生命科学研究和人类医学。添加、删除或编辑人类 DNA 序列的能力为治疗先天性和获得性人类疾病带来了变革性的潜力。细胞和基因治疗生态系统的及时成熟及其与 CRISPR-Cas 技术的无缝集成，使得能够开发出潜在治愈不仅是镰状细胞贫血和肌肉营养不良等单基因疾病，而且还能治愈癌症和糖尿病等复杂异质疾病的疗法。在这里，我们回顾了涉及使用各种 CRISPR-Cas 系统作为人类疾病治疗方法的临床试验的现状，讨论了挑战，并探讨了新的基于 CRISPR-Cas 的工具，如碱基编辑、先导编辑、基于 CRISPR 的转录调控、基于 CRISPR 的表观基因组编辑和 RNA 编辑，每个工具都具有有前景的新功能和更广泛的治疗潜力。最后，我们讨论了 CRISPR-Cas 系统如何通过生成用于新兴疗法临床前测试的大型动物疾病模型来用于了解人类疾病的生物学。
