Université Paris-Saclay, CNRS, Institut Galien Paris-Saclay, 92296 Châtenay-Malabry, France.
Adv Drug Deliv Rev. 2021 Aug;175:113809. doi: 10.1016/j.addr.2021.05.019. Epub 2021 May 24.
Thanks to their abilities to modulate the expression of virtually any genes, RNA therapeutics have attracted considerable research efforts. Among the strategies focusing on nucleic acid gene inhibitors, antisense oligonucleotides and small interfering RNAs have reached advanced clinical trial phases with several of them having recently been marketed. These successes were obtained by overcoming stability and cellular delivery issues using either chemically modified nucleic acids or nanoparticles. As nucleic acid gene inhibitors are promising strategies to treat inflammatory diseases, this review focuses on the barriers, from manufacturing issues to cellular/subcellular delivery, that still need to be overcome to deliver the nucleic acids to sites of inflammation other than the liver. Furthermore, key examples of applications in rheumatoid arthritis, inflammatory bowel, and lung diseases are presented as case studies of systemic, oral, and lung nucleic acid delivery.
由于它们能够调节几乎任何基因的表达,RNA 疗法引起了相当大的研究兴趣。在专注于核酸基因抑制剂的策略中,反义寡核苷酸和小干扰 RNA 已经进入了临床试验的后期阶段,其中一些已经最近上市。这些成功是通过使用化学修饰的核酸或纳米颗粒克服稳定性和细胞递送来实现的。由于核酸基因抑制剂是治疗炎症性疾病的有前途的策略,本综述重点介绍了从制造问题到细胞/亚细胞递送到除肝脏以外的炎症部位传递核酸仍然需要克服的障碍。此外,还介绍了类风湿关节炎、炎症性肠病和肺部疾病的应用的关键实例,作为全身、口服和肺部核酸递送的案例研究。