State Key Laboratory of Natural and Biomimetic Drugs, School of Pharmaceutical Sciences, Peking University, Beijing, China.
Med Res Rev. 2018 May;38(3):829-869. doi: 10.1002/med.21479. Epub 2018 Jan 5.
Gene-based therapy is one of essential therapeutic strategies for precision medicine through targeting specific genes in specific cells of target tissues. However, there still exist many problems that need to be solved, such as safety, stability, selectivity, delivery, as well as immunity. Currently, the key challenges of gene-based therapy for clinical potential applications are the safe and effective nucleic acid drugs as well as their safe and efficient gene delivery systems. In this review, we first focus on current nucleic acid drugs and their formulation in clinical trials and on the market, including antisense oligonucleotide, siRNA, aptamer, and plasmid nucleic acid drugs. Subsequently, we summarize different chemical modifications of nucleic acid drugs as well as their delivery systems for gene-based therapeutics in vivo based on nucleic acid chemistry and nanotechnology methods.
基因治疗是精准医学的重要治疗策略之一，通过针对靶组织中特定细胞的特定基因。然而，仍然存在许多需要解决的问题，如安全性、稳定性、选择性、传递以及免疫原性。目前，基因治疗在临床应用中的关键挑战是安全有效的核酸药物及其安全有效的基因传递系统。在这篇综述中，我们首先关注目前处于临床试验和市场阶段的核酸药物及其制剂，包括反义寡核苷酸、siRNA、适体和质粒核酸药物。随后，我们根据核酸化学和纳米技术方法，总结了不同的核酸药物化学修饰及其体内基因治疗的传递系统。
