Children's Mercy Kansas City, Kansas City, Missouri, USA.
University of Missouri-Kansas City School of Medicine, Kansas City, Missouri, USA.
Clin Transl Sci. 2019 Nov;12(6):573-585. doi: 10.1111/cts.12668. Epub 2019 Aug 5.
RNA interference via small interfering RNA (siRNA) offers opportunities to precisely target genes that contribute to gastrointestinal (GI) pathologies, such as inflammatory bowel disease, celiac, and esophageal scarring. Delivering the siRNA to the GI tract proves challenging as the harsh environment of the intestines degrades the siRNA before it can reach its target or blocks its entry into its site of action in the cytoplasm. Additionally, the GI tract is large and disease is often localized to a specific site. This review discusses polymer and lipid-based delivery systems for protection and targeting of siRNA therapies to the GI tract to treat local disease.
通过小干扰 RNA(siRNA)的 RNA 干扰为靶向导致胃肠道(GI)疾病的基因提供了机会,例如炎症性肠病、乳糜泻和食管瘢痕。将 siRNA 递送到胃肠道具有挑战性,因为肠道恶劣的环境会在 siRNA 到达靶标之前使其降解,或阻止其进入细胞质中的作用部位。此外,胃肠道很大,疾病通常局限于特定部位。这篇综述讨论了聚合物和脂质基递药系统,用于保护和靶向 siRNA 疗法到胃肠道以治疗局部疾病。