越来越多的证据表明，恶性肿瘤是由一小部分具有类似于正常成体干细胞的生物学特性的肿瘤细胞亚群首要形成和维持的。这一极小的肿瘤干细胞（Cancer Stem Cells, CSC）群体组成了导致癌症复发、耐药和转移的肿瘤启动细胞。传统治疗，如化疗、放疗和手术，除了潜在的毒性和非特异性外，反而可能增加CSC的数量、扩散和生存。下一代测序和组学技术正在增加我们对CSC发展所涉及的途径和因素的认识，并有助于发现针对CSC的新治疗靶点。此外，纳米医学的最新进展为开发灭绝CSC的最佳特异性疗法提供了希望。而且，人工智能和纳米信息学的应用可以阐明新的药物靶点，并帮助设计用于应对CSC的药物和纳米颗粒（NPs）。在本综述中，我们首先总结了CSC的特性，并描述了导致CSC的出现和生存的信号通路和分子特征。此外，我们还讨论了CSC在肿瘤内的位置以及宿主因素对CSC的形成和维持的影响。重点介绍了肿瘤干性涉及的新发现的分子靶点以及一些对抗CSC的新型治疗化合物。此外，还对抗CSC纳米颗粒（Anti-CSC NPs）的最佳特性进行了讨论，包括血液循环和稳定性，肿瘤聚集和渗透，细胞内吸收，药物释放，内质网逃逸以及针对CSC的特异性适配体设计。最后，还讨论了一些用于克服CSC的治疗和治疗诊断目的而设计的最新智能纳米颗粒。 © 2023. Elsevier B.V. 发表。

There is increasing evidence that malignant tumors are initiated and maintained by a sub-population of tumor cells that have similar biological properties to normal adult stem cells. This very small population of Cancer Stem Cells (CSC) comprises tumor initiating cells responsible for cancer recurrence, drug resistance and metastasis. Conventional treatments such as chemotherapy, radiotherapy and surgery, in addition to being potentially toxic and non-specific, may paradoxically increase the population, spread and survival of CSCs. Next-generation sequencing and omics technologies are increasing our understanding of the pathways and factors involved in the development of CSCs, and can help to discover new therapeutic targets against CSCs. In addition, recent advances in nanomedicine have provided hope for the development of optimal specific therapies to eradicate CSCs. Moreover, the use of artificial intelligence and nano-informatics can elucidate new drug targets, and help to design drugs and nanoparticles (NPs) to deal with CSCs. In this review, we first summarize the properties of CSCs and describe the signaling pathways and molecular characteristics responsible for the emergence and survival of CSCs. Also, the location of CSCs within the tumor and the effect of host factors on the creation and maintenance of CSCs are discussed. Newly discovered molecular targets involved in cancer stemness and some novel therapeutic compounds to combat CSCs are highlighted. The optimum properties of anti-CSC NPs, including blood circulation and stability, tumor accumulation and penetration, cellular internalization, drug release, endosomal escape, and aptamers designed for specific targeting of CSCs are covered. Finally, some recent smart NPs designed for therapeutic and theranostic purposes to overcome CSCs are discussed.Copyright © 2023. Published by Elsevier B.V.