Severe injuries and diseases of bone tissue caused by congenital disorder, extrinsic trauma, infection, and osteosarcoma resection face significant challenges for complete recovery. The current treatment options with therapeutic molecules (e.g., drugs, proteins, genetic molecules) often involve vector-borne infection, restrictive availability, donor deficiency, and immune rejection, whereas the materials-based nanotherapeutics can tackle these issues. Nanotherapeutics can secure the stability and activity of cargo molecules, allow their controlled release, and even target the specific cells and organelles, all of which eventually improve the in vivo efficacy while relieving the off-target concerns. Here we systematically review the recent progress in materials-based nanotherapeutics for the treatment of injured and diseased bone, highlighting the processing nanotechnologies and delivery strategies of various classes of therapeutic molecules that intervene specific cells and intracellular signaling pathways. Nanotherapeutics combined with three-dimensional matrices are also underscored that can recruit endogenous cells and sequester intrinsic therapeutic molecules, ultimately supporting and boosting the microenvironment for regenerative process. Future aspects in the development of nanotherapeutics approach and clinical availability are also discussed.

先天性疾病、外伤、感染、骨肉瘤切除等引起的骨组织严重损伤和疾病，面临完全康复的重大挑战。当前使用治疗性分子（例如药物、蛋白质、遗传分子）的治疗选择通常涉及媒介传播感染、限制可用性、供体缺陷和免疫排斥，而基于材料的纳米疗法可以解决这些问题。纳米疗法可以确保货物分子的稳定性和活性，允许它们受控释放，甚至靶向特定的细胞和细胞器，所有这些最终都会改善体内在缓解脱靶问题的同时提高疗效。在这里，我们系统地回顾了用于治疗受伤和患病骨骼的基于材料的纳米疗法的最新进展，重点介绍了干预特定细胞和细胞内信号通路的各类治疗分子的加工纳米技术和递送策略。还强调了纳米治疗与三维矩阵相结合，可以募集内源性细胞并隔离内在治疗分子，最终支持和促进再生过程的微环境。还讨论了纳米治疗方法和临床可用性发展的未来方面。