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使用负载有小干扰RNA的可生物降解聚合物纳米颗粒进行阴道内基因沉默。

Intravaginal gene silencing using biodegradable polymer nanoparticles densely loaded with small-interfering RNA.

作者信息

Woodrow Kim A, Cu Yen, Booth Carmen J, Saucier-Sawyer Jennifer K, Wood Monica J, Saltzman W Mark

机构信息

Department of Biomedical Engineering, Yale University, New Haven, Connecticut 06511, USA.

出版信息

Nat Mater. 2009 Jun;8(6):526-33. doi: 10.1038/nmat2444. Epub 2009 May 3.

Abstract

Vaginal instillation of small-interfering RNA (siRNA) using liposomes has led to silencing of endogenous genes in the genital tract and protection against challenge from infectious disease. Although siRNA lipoplexes are easily formulated, several of the most effective transfection agents available commercially may be toxic to the mucosal epithelia and none are able to provide controlled or sustained release. Here, we demonstrate an alternative approach using nanoparticles composed entirely of FDA-approved materials. To render these materials effective for gene silencing, we developed novel approaches to load them with high amounts of siRNA. A single dose of siRNA-loaded nanoparticles to the mouse female reproductive tract caused efficient and sustained gene silencing. Knockdown of gene expression was observed proximal (in the vaginal lumen) and distal (in the uterine horns) to the site of topical delivery. In addition, nanoparticles penetrated deep into the epithelial tissue. This is the first report demonstrating that biodegradable polymer nanoparticles are effective delivery vehicles for siRNA to the vaginal mucosa.

摘要

使用脂质体进行阴道内小干扰RNA(siRNA)给药已导致生殖道内源性基因沉默,并提供针对传染病攻击的保护作用。尽管siRNA脂质复合物易于制备,但市售的几种最有效的转染剂可能对粘膜上皮有毒性,并且没有一种能够提供可控或持续释放。在此,我们展示了一种使用完全由美国食品药品监督管理局(FDA)批准的材料组成的纳米颗粒的替代方法。为使这些材料对基因沉默有效,我们开发了将大量siRNA加载到其中的新方法。向小鼠雌性生殖道单次给药负载siRNA的纳米颗粒可导致高效且持续的基因沉默。在局部给药部位的近端(阴道腔内)和远端(子宫角)均观察到基因表达的敲低。此外,纳米颗粒深入渗透到上皮组织中。这是第一份证明可生物降解的聚合物纳米颗粒是将siRNA递送至阴道粘膜的有效载体的报告。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2fc8/2693358/4741eccd796b/nihms-108744-f0001.jpg

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