Jump to main content
Jump to site search

Issue 11, 2020
Previous Article Next Article

Polyornithine-based polyplexes to boost effective gene silencing in CNS disorders

Author affiliations

Abstract

Gene silencing therapies have successfully suppressed the translation of target proteins, a strategy that holds great promise for the treatment of central nervous system (CNS) disorders. Advances in the current knowledge on multimolecular delivery vehicles are concentrated on overcoming the difficulties in delivery of small interfering (si)RNA to target tissues, which include anatomical accessibility, slow diffusion, safety concerns, and the requirement for specific cell uptake within the unique environment of the CNS. The present work addressed these challenges through the implementation of polyornithine derivatives in the construction of polyplexes used as non-viral siRNA delivery vectors. Physicochemical and biological characterization revealed biodegradability and biocompatibility of our polyornithine-based system and the ability to silence gene expression in primary oligodendrocyte progenitor cells (OPCs) effectively. In summary, the well-defined properties and neurological compatibility of this polypeptide-based platform highlight its potential utility in the treatment of CNS disorders.

Graphical abstract: Polyornithine-based polyplexes to boost effective gene silencing in CNS disorders

Back to tab navigation

Supplementary files

Article information


Submitted
20 Jul 2019
Accepted
02 Dec 2019
First published
06 Dec 2019

Nanoscale, 2020,12, 6285-6299
Article type
Paper

Polyornithine-based polyplexes to boost effective gene silencing in CNS disorders

I. Conejos-Sánchez, E. Gallon, A. Niño-Pariente, J. A. Smith, A. G. De la Fuente, L. Di Canio, S. Pluchino, R. J. M. Franklin and M. J. Vicent, Nanoscale, 2020, 12, 6285
DOI: 10.1039/C9NR06187H

Social activity

Search articles by author

Spotlight

Advertisements