Nanotechnology for in vivo targeted siRNA delivery

James E Dahlman; Kevin J Kauffman; Robert Langer; Daniel G Anderson

doi:10.1016/B978-0-12-800148-6.00003-1

Nanotechnology for in vivo targeted siRNA delivery

Adv Genet. 2014:88:37-69. doi: 10.1016/B978-0-12-800148-6.00003-1.

Authors

James E Dahlman¹, Kevin J Kauffman², Robert Langer³, Daniel G Anderson³

Affiliations

¹ The Institute for Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, MA, USA; Harvard-MIT Division of Health Sciences and Technology, Massachusetts Institute of Technology, Cambridge, MA, USA; David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA.
² Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA; David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA.
³ The Institute for Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, MA, USA; Harvard-MIT Division of Health Sciences and Technology, Massachusetts Institute of Technology, Cambridge, MA, USA; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA; David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA.

PMID: 25409603
DOI: 10.1016/B978-0-12-800148-6.00003-1

Abstract

Small interfering RNAs (siRNAs) can specifically inhibit gene expression. As a result, they have tremendous scientific and clinical potential. However, the use of these molecules in patients and animal models has been limited by challenges with delivery. Intracellular RNA delivery is difficult; it requires a system that protects the siRNA from degradative nucleases in the bloodstream, minimizes clearance by the reticuloendothelial system, maximizes delivery to the target tissue, and promotes entry into, and out of, an endocytic vesicle. Despite these barriers, recent data suggest that RNA may be targeted to cells of interest in vivo. Herein we outline strategies for targeted siRNA delivery, and describe how these strategies may be improved.

Keywords: Drug delivery; Lipid; Lipidoid; Nanoparticle; Nanotechnology; miRNA; siRNA.

Publication types

Review

MeSH terms

Animals
Drug Delivery Systems
Endothelial Cells / drug effects
Gene Expression Regulation
Gene Transfer Techniques
Humans
Lipids / administration & dosage
Lipids / chemistry
Liver / drug effects
Liver / physiology*
Nanoparticles / administration & dosage
Nanoparticles / chemistry*
Nanotechnology / methods*
Neoplasms / genetics
Neoplasms / pathology
RNA, Small Interfering / administration & dosage*

Substances

Lipids
RNA, Small Interfering