过去十年，我们对Hedgehog (HH)信号通路在各种生物学事件中的理解取得了重大进展。HH信号通路通过涉及原生纤毛的复杂信号级联发挥其生物学效应。HH信号通路在胚胎发育和组织稳态中具有重要功能。它在调节成年干细胞的增殖和分化方面起到了核心作用。重要的是，越来越明确的是，HH信号通路与癌症患病率的增加、恶性进展、恶劣预后甚至增加的死亡率有关。了解HH信号通路的综合性质为癌症的新治疗靶点开辟了潜力。已经开发出各种药物，包括小分子抑制剂、天然化合物和长非编码RNA (LncRNA)，其中一些已经获得了临床批准。本综述概述了HH信号在组织稳态和癌症中的最新发现，并讨论这些进展如何为基于生物学的癌症治疗的发展铺平道路。此外，我们还讨论了HH信号通路靶向治疗的现状和限制。本综述的观点将有助于读者了解HH信号在稳态和癌症中的功能，以及癌症治疗靶点的机遇和挑战。© 2023. 四川大学华西医院。

The past decade has seen significant advances in our understanding of Hedgehog (HH) signaling pathway in various biological events. HH signaling pathway exerts its biological effects through a complex signaling cascade involved with primary cilium. HH signaling pathway has important functions in embryonic development and tissue homeostasis. It plays a central role in the regulation of the proliferation and differentiation of adult stem cells. Importantly, it has become increasingly clear that HH signaling pathway is associated with increased cancer prevalence, malignant progression, poor prognosis and even increased mortality. Understanding the integrative nature of HH signaling pathway has opened up the potential for new therapeutic targets for cancer. A variety of drugs have been developed, including small molecule inhibitors, natural compounds, and long non-coding RNA (LncRNA), some of which are approved for clinical use. This review outlines recent discoveries of HH signaling in tissue homeostasis and cancer and discusses how these advances are paving the way for the development of new biologically based therapies for cancer. Furthermore, we address status quo and limitations of targeted therapies of HH signaling pathway. Insights from this review will help readers understand the function of HH signaling in homeostasis and cancer, as well as opportunities and challenges of therapeutic targets for cancer.© 2023. West China Hospital, Sichuan University.