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Current Medicinal Chemistry


ISSN (Print): 0929-8673
ISSN (Online): 1875-533X

Endosome Escape Strategies for Improving the Efficacy of Oligonucleotide Delivery Systems

Author(s): Y. Wan, P.M. Moyle and I. Toth

Volume 22 , Issue 29 , 2015

Page: [3326 - 3346] Pages: 21

DOI: 10.2174/0929867322666150825162941

Price: $65


Gene therapy requires safe and effective vectors to deliver genes to their target site of action. Non-viral gene delivery systems have attracted growing attention due to their low toxicity, low immunogenicity and ease of production compared to viral vectors. Most non-viral gene delivery systems enter cells via endocytic pathways, and their escape from endosomes is therefore crucial for successful transfection. Several reagents have been developed to promote endosomal escape, including peptides, polymers and lipids. Among these, endosome-disrupting peptides have been used in many studies, and have proven to be one of the most promising approaches to overcome endosomal entrapment and lysosomal degradation. This review provides an up-to-date summary of strategies for enhancing endosomal escape, with a focus on the modification of endosome-disrupting peptides to further increase the efficient delivery of oligonucleotides.

Keywords: Endosome escape, fusogenic peptides, gene delivery, non-viral vectors, oligonucleotides, peptides.

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