Water-soluble substituted chitosan derivatives as technology platform for inhalation delivery of siRNA

Despite research efforts full potential of siRNA-based therapeutics has not yet been fully realized due to a need for suitable, effective delivery formulations. Here, we examine a potential of a new class of water-soluble chitosans as siRNA platform for pulmonary delivery. The system is based on pip...

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Main Authors: Capel, Victoria, Vllasaliu, Driton, Watts, Peter, Clarke, Philip A., Luxton, Dominic, Grabowska, Anna M., Mantovani, Giuseppe, Stolnik, Snow
Format: Article
Language:English
Published: Taylor & Francis 2018
Subjects:
Online Access:https://eprints.nottingham.ac.uk/50532/
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author Capel, Victoria
Vllasaliu, Driton
Watts, Peter
Clarke, Philip A.
Luxton, Dominic
Grabowska, Anna M.
Mantovani, Giuseppe
Stolnik, Snow
author_facet Capel, Victoria
Vllasaliu, Driton
Watts, Peter
Clarke, Philip A.
Luxton, Dominic
Grabowska, Anna M.
Mantovani, Giuseppe
Stolnik, Snow
author_sort Capel, Victoria
building Nottingham Research Data Repository
collection Online Access
description Despite research efforts full potential of siRNA-based therapeutics has not yet been fully realized due to a need for suitable, effective delivery formulations. Here, we examine a potential of a new class of water-soluble chitosans as siRNA platform for pulmonary delivery. The system is based on piperazine-substituted chitosans, a material designed to integrate established, safe application of chitosan for mucosal administration with novel properties: the piperazine-substituted chitosans are freely water-soluble at physiological pH, possess low cytotoxicity (no significant reduction in cell viability up to 0.1 mg/ml), and provide efficient incorporation of siRNA into sub-300 nm colloidal complexes (at relatively low polymer/siRNA ratio of 5:1). In vitro, the complexes achieved silencing of a model gene at a level of 40–80%, when tested in a panel of lung epithelial cells. Considering the formulation ‘developability’, there were no significant changes in the complexes’ size and integrity on aerosolisation by microsprayer (PenCenturyTM) device. Following intratracheal aerolisation, the complexes deposited throughout the lung, although relatively inhomogeneously, as judged from IVIS imaging of the isolated mouse lung (visualizing DY647-siRNA). In vivo data illustrate absence of adverse effects on repeated administration of complexes and significant tumor reduction in atopical lung cancer model in mice. Altogether, the data illustrates potential of substituted chitosan derivatives to be utilized as a safe system for inhalation delivery of siRNA.
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spelling nottingham-505322018-03-20T11:54:02Z https://eprints.nottingham.ac.uk/50532/ Water-soluble substituted chitosan derivatives as technology platform for inhalation delivery of siRNA Capel, Victoria Vllasaliu, Driton Watts, Peter Clarke, Philip A. Luxton, Dominic Grabowska, Anna M. Mantovani, Giuseppe Stolnik, Snow Despite research efforts full potential of siRNA-based therapeutics has not yet been fully realized due to a need for suitable, effective delivery formulations. Here, we examine a potential of a new class of water-soluble chitosans as siRNA platform for pulmonary delivery. The system is based on piperazine-substituted chitosans, a material designed to integrate established, safe application of chitosan for mucosal administration with novel properties: the piperazine-substituted chitosans are freely water-soluble at physiological pH, possess low cytotoxicity (no significant reduction in cell viability up to 0.1 mg/ml), and provide efficient incorporation of siRNA into sub-300 nm colloidal complexes (at relatively low polymer/siRNA ratio of 5:1). In vitro, the complexes achieved silencing of a model gene at a level of 40–80%, when tested in a panel of lung epithelial cells. Considering the formulation ‘developability’, there were no significant changes in the complexes’ size and integrity on aerosolisation by microsprayer (PenCenturyTM) device. Following intratracheal aerolisation, the complexes deposited throughout the lung, although relatively inhomogeneously, as judged from IVIS imaging of the isolated mouse lung (visualizing DY647-siRNA). In vivo data illustrate absence of adverse effects on repeated administration of complexes and significant tumor reduction in atopical lung cancer model in mice. Altogether, the data illustrates potential of substituted chitosan derivatives to be utilized as a safe system for inhalation delivery of siRNA. Taylor & Francis 2018-03-01 Article PeerReviewed application/pdf en cc_by https://eprints.nottingham.ac.uk/50532/1/Water%20soluble%20substituted%20chitosan%20derivatives%20as%20technology%20platform%20for%20inhalation%20delivery%20of%20siRNA.pdf Capel, Victoria, Vllasaliu, Driton, Watts, Peter, Clarke, Philip A., Luxton, Dominic, Grabowska, Anna M., Mantovani, Giuseppe and Stolnik, Snow (2018) Water-soluble substituted chitosan derivatives as technology platform for inhalation delivery of siRNA. Drug Delivery, 25 (1). pp. 644-653. ISSN 1521-0464 Chitosan siRNA delivery siRNA complex lung inhalation IVIS imaging https://www.tandfonline.com/doi/full/10.1080/10717544.2018.1440668 doi:10.1080/10717544.2018.1440668 doi:10.1080/10717544.2018.1440668
spellingShingle Chitosan
siRNA delivery
siRNA complex
lung inhalation
IVIS imaging
Capel, Victoria
Vllasaliu, Driton
Watts, Peter
Clarke, Philip A.
Luxton, Dominic
Grabowska, Anna M.
Mantovani, Giuseppe
Stolnik, Snow
Water-soluble substituted chitosan derivatives as technology platform for inhalation delivery of siRNA
title Water-soluble substituted chitosan derivatives as technology platform for inhalation delivery of siRNA
title_full Water-soluble substituted chitosan derivatives as technology platform for inhalation delivery of siRNA
title_fullStr Water-soluble substituted chitosan derivatives as technology platform for inhalation delivery of siRNA
title_full_unstemmed Water-soluble substituted chitosan derivatives as technology platform for inhalation delivery of siRNA
title_short Water-soluble substituted chitosan derivatives as technology platform for inhalation delivery of siRNA
title_sort water-soluble substituted chitosan derivatives as technology platform for inhalation delivery of sirna
topic Chitosan
siRNA delivery
siRNA complex
lung inhalation
IVIS imaging
url https://eprints.nottingham.ac.uk/50532/
https://eprints.nottingham.ac.uk/50532/
https://eprints.nottingham.ac.uk/50532/