Polyornithine-based polyplexes to boost effective gene silencing in CNS disorders.
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Authors
Conejos-Sánchez, I
Gallon, E
Niño-Pariente, A
Smith, JA
De la Fuente, AG
Di Canio, L
Vicent, MJ
Publication Date
2019-12-16Journal Title
Nanoscale
ISSN
2040-3364
Publisher
Royal Society of Chemistry
Language
eng
Type
Article
This Version
AM
Metadata
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Conejos-Sánchez, I., Gallon, E., Niño-Pariente, A., Smith, J., De la Fuente, A., Di Canio, L., Pluchino, S., et al. (2019). Polyornithine-based polyplexes to boost effective gene silencing in CNS disorders.. Nanoscale https://doi.org/10.1039/c9nr06187h
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.
Sponsorship
MRC (MC_PC_12009)
Identifiers
External DOI: https://doi.org/10.1039/c9nr06187h
This record's URL: https://www.repository.cam.ac.uk/handle/1810/301251
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