Singh Puneetpal, Singh Monica, Singh Baani, Sharma Kirti, Kumar Nitin, Singh Deepinder, Klair Harpal Singh, Mastana Sarabjit
Department of Human Genetics, Punjabi University, Patiala 147002, Punjab, India.
Vardhman Mahavir Health Care, Urban Estate, Ph-II, Patiala 147002, Punjab, India.
Biomedicines. 2024 Jan 1;12(1):90. doi: 10.3390/biomedicines12010090.
The global statistics of bone disorders, skeletal defects, and fractures are frightening. Several therapeutic strategies are being used to fix them; however, RNAi-based siRNA therapy is starting to prove to be a promising approach for the prevention of bone disorders because of its advanced capabilities to deliver siRNA or siRNA drug conjugate to the target tissue. Despite its 'bench-to-bedside' usefulness and approval by food and drug administration for five siRNA-based therapeutic medicines: Patisiran, Vutrisiran, Inclisiran, Lumasiran, and Givosiran, its use for the other diseases still remains to be resolved. By correcting the complications and complexities involved in siRNA delivery for its sustained release, better absorption, and toxicity-free activity, siRNA therapy can be harnessed as an experimental tool for the prevention of complex and undruggable diseases with a personalized medicine approach. The present review summarizes the findings of notable research to address the implications of siRNA in bone health for the restoration of bone mass, recovery of bone loss, and recuperation of bone fractures.
全球骨骼疾病、骨骼缺陷和骨折的统计数据令人担忧。目前正在采用多种治疗策略来解决这些问题;然而,基于RNA干扰的小干扰RNA(siRNA)疗法正开始被证明是一种预防骨骼疾病的有前景的方法,因为它具有将siRNA或siRNA药物偶联物递送至靶组织的先进能力。尽管其具有“从实验室到临床”的实用性,并且已获得食品药品监督管理局对五种基于siRNA的治疗药物(帕替拉韦、维曲西泮、英克西兰、鲁马西泮和吉沃西兰)的批准,但其在其他疾病中的应用仍有待解决。通过纠正siRNA递送过程中涉及的并发症和复杂性,以实现其持续释放、更好的吸收和无毒性活性,siRNA疗法可作为一种实验工具,采用个性化医疗方法预防复杂且难以药物治疗的疾病。本综述总结了显著研究的结果,以探讨siRNA在骨骼健康方面对恢复骨量、恢复骨质流失和修复骨折的影响。