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ICS - 283:一种用于小干扰RNA靶向静脉内递送的系统。

ICS-283: a system for targeted intravenous delivery of siRNA.

作者信息

Schiffelers Raymond M, Storm Gert

机构信息

Department of Pharmaceutics, Room Z735A, Utrecht Institute for Pharmaceutical Sciences, PO Box 80082, 3508 TB, Utrecht, The Netherlands.

出版信息

Expert Opin Drug Deliv. 2006 May;3(3):445-54. doi: 10.1517/17425247.3.3.445.

Abstract

ICS-283 was developed within Intradigm Corporation as a system that is designed for the systemic delivery of therapeutic small interfering (siRNA) to sites of pathological angiogenesis. The non-viral siRNA delivery system is based on synthetic nanoparticles, known as Targe (Intradigm Corporation), which functions as a broad-platform technology to deliver siRNA to specific target cells in diseased tissues. The system is constructed to incorporate different functionalities that address critical needs for successful nucleic acid delivery. The TargeTran synthetic vector is a self-assembling, layered nanoparticle that protects and targets siRNA to specific cell types in pathological tissues. At present, ICS-283 is the only antiangiogenic siRNA delivery system that is designed for intravenous administration to treat angiogenesis-driven diseases.

摘要

ICS-283是由Intradigm公司研发的一种系统,旨在将治疗性小干扰RNA(siRNA)全身性递送至病理性血管生成部位。这种非病毒siRNA递送系统基于名为Targe(Intradigm公司)的合成纳米颗粒,它作为一种广泛应用的平台技术,可将siRNA递送至患病组织中的特定靶细胞。该系统的构建整合了不同功能,以满足成功进行核酸递送的关键需求。TargeTran合成载体是一种自组装的层状纳米颗粒,可保护siRNA并将其靶向病理性组织中的特定细胞类型。目前,ICS-283是唯一一种设计用于静脉内给药以治疗血管生成驱动疾病的抗血管生成siRNA递送系统。

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