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Polyethylenimines for siRNA and miRNA delivery in vivo
Author(s) -
Höbel Sabrina,
Aigner Achim
Publication year - 2013
Publication title -
wiley interdisciplinary reviews: nanomedicine and nanobiotechnology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.175
H-Index - 72
eISSN - 1939-0041
pISSN - 1939-5116
DOI - 10.1002/wnan.1228
Subject(s) - small interfering rna , nanomedicine , microrna , gene knockdown , rna interference , in vivo , rna , drug discovery , drug delivery , gene silencing , biology , computational biology , chemistry , bioinformatics , nanotechnology , gene , biochemistry , materials science , microbiology and biotechnology , nanoparticle
The discovery of RNA interference ( RNAi ) as a naturally occurring mechanism for gene knockdown has attracted considerable attention toward the use of small interfering RNAs ( siRNAs ) for therapeutic purposes. Likewise, microRNAs ( miRNAs ) have emerged as important cellular regulators of gene expression, and their pathological underexpression allows for novel therapeutic strategies (‘ miRNA replacement therapy’). To address issues related to the instability, charge, and molecular weight of small RNA molecules, nanoparticle formulations have been explored for their in vivo application. Polyethylenimines (PEIs) are positively charged, linear, or branched polymers that are able to form nanoscale complexes with small RNAs , leading to RNA protection, cellular delivery, and intracellular release. This review highlights the important properties of various PEIs with regard to their use for in vivo RNA delivery. PEI modifications for increased efficacy, altered pharmacokinetic properties, improved biocompatibility and, upon covalent coupling of ligands, targeted delivery are described. An overview of various modified PEIs and a comprehensive list of representative studies using PEI ‐based siRNA or miRNA delivery in vivo are given. WIREs Nanomed Nanobiotechnol 2013. doi: 10.1002/wnan.1228 This article is categorized under: Therapeutic Approaches and Drug Discovery > Emerging Technologies Therapeutic Approaches and Drug Discovery > Nanomedicine for Neurological Disease Therapeutic Approaches and Drug Discovery > Nanomedicine for Oncologic Disease