Fernández Fernández, E, Santos-Carballal, B, Weber, WM et al. (1 more author) (2016) Chitosan as a non-viral co-transfection system in a cystic fibrosis cell line. International Journal of Pharmaceutics, 502 (1-2). pp. 1-9. ISSN 0378-5173
Abstract
Successful gene therapy requires the development of suitable vehicles for the selective and efficient delivery of genes to specific target cells at the expense of minimal toxicity. In this work, we investigated a non-viral gene delivery system based on chitosan (CS) to specifically address cystic fibrosis (CF). Thus, electrostatic self-assembled CS-pEGFP and CS-pEGFP-siRNA complexes were prepared from high-pure fully characterized CS (Mw ∼ 20 kDa and degree of acetylation ∼ 30%). The average diameter of positively-charged complexes (i.e. ζ ∼+25 mV) was ∼ 200 nm. The complexes were found relatively stable over 14h in Opti-MEM. Cell viability study did not show any significant cytotoxic effect of the CS-based complexes in a human bronchial cystic fibrosis cell line (CFBE41o-). We evaluated the transfection efficiency of this cell line with both CS-pEGFP and co-transfected with CS-pEGFP-siRNA complexes at (N/P) charge ratio of 12. We reported an increase in the fluorescence intensity of CS-pEGFP and a reduction in the cells co-transfected with CS-pEGFP-siRNA. This study shows proof-of-principle that co-transfection with chitosan might be an effective delivery system in a human CF cell line. It also offers a potential alternative to further develop therapeutic strategies for inherited disease treatments, such as CF.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © 2016, Elsevier B.V. This is an author produced version of a paper published in International Journal of Pharmaceutics. Uploaded in accordance with the publisher's self-archiving policy. |
Keywords: | Chitosan; Cystic fibrosis; Gene delivery; Nanoparticles; Transfection |
Dates: |
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Institution: | The University of Leeds |
Academic Units: | The University of Leeds > Faculty of Environment (Leeds) > School of Food Science and Nutrition (Leeds) |
Depositing User: | Symplectic Publications |
Date Deposited: | 24 Nov 2016 12:59 |
Last Modified: | 11 Apr 2017 02:54 |
Published Version: | https://doi.org/10.1016/j.ijpharm.2016.01.083 |
Status: | Published |
Publisher: | Elsevier |
Identification Number: | 10.1016/j.ijpharm.2016.01.083 |
Related URLs: | |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:107203 |