Materials 2018, 11(1), 122; https://doi.org/10.3390/ma11010122
Biopolymer-Based Nanoparticles for Cystic Fibrosis Lung Gene Therapy Studies
Elena Fernández Fernández 1,*
, Beatriz Santos-Carballal 2, Chiara de Santi 1, Joanne M. Ramsey 3, Ronan MacLoughlin 3,4,5, Sally-Ann Cryan 3 and Catherine M. Greene 1
, Beatriz Santos-Carballal 2, Chiara de Santi 1, Joanne M. Ramsey 3, Ronan MacLoughlin 3,4,5, Sally-Ann Cryan 3 and Catherine M. Greene 11
Lung Biology Group, Department of Clinical Microbiology, Royal College of Surgeons in Ireland, Beaumont Hospital, Dublin 9, Ireland
2
ChiPro GmbH, Anne-Conway-Straße 1, 28359 Bremen, Germany
3
School of Pharmacy, Royal College of Surgeons in Ireland, Dublin 2, Ireland
4
School of Pharmacy and Pharmaceutical Sciences, Trinity College, Dublin 2, Ireland
5
Aerogen Ltd., Galway Business Park, Dangan, Galway H91 HE94, Ireland
*
Author to whom correspondence should be addressed.
Received: 30 November 2017 / Revised: 4 January 2018 / Accepted: 8 January 2018 / Published: 13 January 2018
(This article belongs to the Special Issue Nanomaterials for Biomedical Applications)
Abstract
Lung gene therapy for cystic fibrosis disease has not been successful due to several challenges such as the absence of an appropriate vector. Therefore, optimal delivery of emerging therapeutics to airway epithelial cells demands suitable non-viral systems. In this work, we describe the formulation and the physicochemical investigation of biocompatible and biodegradable polymeric nanoparticles (NPs), including PLGA and chitosan (animal and non-animal), as novel methods for the safe and efficient delivery of CFTR-specific locked nucleic acids (LNAs). View Full-TextKeywords:
cystic fibrosis; cystic fibrosis transmembrane conductance regulator (CFTR); lung gene delivery; nanoparticles; biopolymers; PLGA; chitosan; Locked-Nucleic Acid (LNA)
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).
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Fernández Fernández, E.; Santos-Carballal, B.; de Santi, C.; Ramsey, J.M.; MacLoughlin, R.; Cryan, S.-A.; Greene, C.M. Biopolymer-Based Nanoparticles for Cystic Fibrosis Lung Gene Therapy Studies. Materials 2018, 11, 122.
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