Sun Xi, Guo Qi, Wei Wenhua, Robertson Stephen, Yuan Ying, Luo Xianghang
Department of Endocrinology, The Third Xiangya Hospital of Central South University, 138# Tongzipo Road, Changsha, Hunan 410007, China.
Department of Endocrinology, Endocrinology Research Center, Xiangya Hospital of Central South University, 87# Xiangya Road, Changsha, Hunan 410008, China.
Int J Endocrinol. 2019 Mar 6;2019:6782653. doi: 10.1155/2019/6782653. eCollection 2019.
Emerging evidence demonstrates that microRNAs, as important endogenous posttranscriptional regulators, are essential for bone remodeling and regeneration. Undoubtedly, microRNA-based gene therapies show great potential to become novel approaches against bone-related diseases, including osteoporosis and associated fractures. The major obstacles for continued advancement of microRNA-based therapies in clinical application include their poor stability, nonspecific biodistribution, and unwanted side effects. Appropriate chemical modifications and delivery vectors, which improve the biological performance and potency of microRNA-based drugs, hold the key to translating miRNA technologies into clinical practice. Thus, this review summarizes the current attempts and existing deficiencies of chemical modifications and delivery systems applied in microRNA-based therapies for osteoporosis and osteoporotic fractures to inform further explorations.
新出现的证据表明,作为重要的内源性转录后调节因子,微小RNA对骨重塑和再生至关重要。毫无疑问,基于微小RNA的基因疗法具有巨大潜力,有望成为治疗包括骨质疏松症及相关骨折在内的骨相关疾病的新方法。基于微小RNA的疗法在临床应用中持续推进的主要障碍包括其稳定性差、生物分布非特异性以及不良副作用。合适的化学修饰和递送载体可改善基于微小RNA的药物的生物学性能和效力,是将微小RNA技术转化为临床实践的关键。因此,本综述总结了目前在基于微小RNA的骨质疏松症和骨质疏松性骨折治疗中应用的化学修饰和递送系统的尝试及存在的不足,以为进一步探索提供参考。