MOE Key Laboratory of Gene Function and Regulation, State Key Laboratory of Biocontrol, School of Life Sciences, Sun Yat-sen University, Guangzhou, Guangdong 510275, China.
Center for Reproductive Medicine, The Third Affiliated Hospital of Sun Yat-sen University, Sun Yat-sen University, Guangzhou, Guangdong 510630, China.
J Genet Genomics. 2024 Feb;51(2):159-183. doi: 10.1016/j.jgg.2023.07.007. Epub 2023 Jul 27.
Osteoarthritis (OA) is one of the most common degenerative joint diseases worldwide, causing pain, disability, and decreased quality of life. The balance between regeneration and inflammation-induced degradation results in multiple etiologies and complex pathogenesis of OA. Currently, there is a lack of effective therapeutic strategies for OA treatment. With the development of CRISPR-based genome, epigenome, and RNA editing tools, OA treatment has been improved by targeting genetic risk factors, activating chondrogenic elements, and modulating inflammatory regulators. Supported by cell therapy and in vivo delivery vectors, genome, epigenome, and RNA editing tools may provide a promising approach for personalized OA therapy. This review summarizes CRISPR-based genome, epigenome, and RNA editing tools that can be applied to the treatment of OA and provides insights into the development of CRISPR-based therapeutics for OA treatment. Moreover, in-depth evaluations of the efficacy and safety of these tools in human OA treatment are needed.
骨关节炎（OA）是全球最常见的退行性关节疾病之一，可导致疼痛、残疾和生活质量下降。再生与炎症诱导的降解之间的平衡导致 OA 的多种病因和复杂发病机制。目前，OA 的治疗缺乏有效的治疗策略。随着基于 CRISPR 的基因组、表观基因组和 RNA 编辑工具的发展，通过针对遗传风险因素、激活软骨形成元件和调节炎症调节剂，OA 的治疗得到了改善。细胞治疗和体内递送载体的支持下，基因组、表观基因组和 RNA 编辑工具可能为 OA 的个性化治疗提供一种有前景的方法。本综述总结了可应用于 OA 治疗的基于 CRISPR 的基因组、表观基因组和 RNA 编辑工具，并探讨了基于 CRISPR 的治疗 OA 的治疗方法的发展。此外，还需要对这些工具在人类 OA 治疗中的疗效和安全性进行深入评估。
