用于寡核苷酸治疗的小干扰RNA的局部和全身递送

Local and systemic delivery of siRNAs for oligonucleotide therapy.

作者信息

Takeshita Fumitaka, Hokaiwado Naomi, Honma Kimi, Banas Agnieszka, Ochiya Takahiro

机构信息

Section for Studies on Metastasis, National Cancer Center Research Institute, Tokyo, Japan.

出版信息

Methods Mol Biol. 2009;487:83-92. doi: 10.1007/978-1-60327-547-7_4.

DOI:10.1007/978-1-60327-547-7_4

PMID:19301643

Abstract

RNA interference (RNAi) is a relatively new found phenomenon of posttranscriptional gene silencing to regulate the expression of multiple genes involved in a wide range of biological processes. The gene-silencing technology via RNAi has also been developed into a commonly anti-gene method. Furthermore, in vivo data indicate that small interfering RNAs (siRNAs) may be used to treat human diseases. However, the most challenging issue to a successful in vivo application is the development of a delivery system that can transport siRNA molecules into the tissues and/or the cells of interest. Also, the evaluation of siRNA potency in vivo is central for the selection of therapeutic siRNAs. In this chapter, the effects of atelocollagen-delivered siRNAs in live animals were monitored using bioluminescence imaging.

摘要

RNA干扰（RNAi）是一种相对较新发现的转录后基因沉默现象，用于调节参与广泛生物学过程的多个基因的表达。通过RNAi的基因沉默技术也已发展成为一种常用的抗基因方法。此外，体内数据表明，小干扰RNA（siRNA）可用于治疗人类疾病。然而，成功进行体内应用最具挑战性的问题是开发一种能够将siRNA分子转运到目标组织和/或细胞中的递送系统。此外，体内siRNA效力的评估对于治疗性siRNA的选择至关重要。在本章中，使用生物发光成像监测了脱细胞胶原蛋白递送的siRNA在活体动物中的作用。