Biopolymer-Based Nanoparticles for Cystic Fibrosis Lung Gene Therapy Studies

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...

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Main Authors:	Elena Fernández Fernández, Beatriz Santos-Carballal, Chiara de Santi, Joanne M. Ramsey, Ronan MacLoughlin, Sally-Ann Cryan, Catherine M. Greene
Format:	Article
Language:	English
Published:	MDPI AG 2018-01-01
Series:	Materials
Subjects:	cystic fibrosis cystic fibrosis transmembrane conductance regulator (CFTR) lung gene delivery nanoparticles biopolymers PLGA chitosan Locked-Nucleic Acid (LNA)
Online Access:	http://www.mdpi.com/1996-1944/11/1/122

id	doaj-f7f63dc0e93b44f7bd5a1c7a579bb38f
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spelling	doaj-f7f63dc0e93b44f7bd5a1c7a579bb38f2020-11-24T22:26:04ZengMDPI AGMaterials1996-19442018-01-0111112210.3390/ma11010122ma11010122Biopolymer-Based Nanoparticles for Cystic Fibrosis Lung Gene Therapy StudiesElena Fernández Fernández0Beatriz Santos-Carballal1Chiara de Santi2Joanne M. Ramsey3Ronan MacLoughlin4Sally-Ann Cryan5Catherine M. Greene6Lung Biology Group, Department of Clinical Microbiology, Royal College of Surgeons in Ireland, Beaumont Hospital, Dublin 9, IrelandChiPro GmbH, Anne-Conway-Straße 1, 28359 Bremen, GermanyLung Biology Group, Department of Clinical Microbiology, Royal College of Surgeons in Ireland, Beaumont Hospital, Dublin 9, IrelandSchool of Pharmacy, Royal College of Surgeons in Ireland, Dublin 2, IrelandSchool of Pharmacy, Royal College of Surgeons in Ireland, Dublin 2, IrelandSchool of Pharmacy, Royal College of Surgeons in Ireland, Dublin 2, IrelandLung Biology Group, Department of Clinical Microbiology, Royal College of Surgeons in Ireland, Beaumont Hospital, Dublin 9, IrelandLung 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).http://www.mdpi.com/1996-1944/11/1/122cystic fibrosiscystic fibrosis transmembrane conductance regulator (CFTR)lung gene deliverynanoparticlesbiopolymersPLGAchitosanLocked-Nucleic Acid (LNA)
collection	DOAJ
language	English
format	Article
sources	DOAJ
author	Elena Fernández Fernández Beatriz Santos-Carballal Chiara de Santi Joanne M. Ramsey Ronan MacLoughlin Sally-Ann Cryan Catherine M. Greene
spellingShingle	Elena Fernández Fernández Beatriz Santos-Carballal Chiara de Santi Joanne M. Ramsey Ronan MacLoughlin Sally-Ann Cryan Catherine M. Greene Biopolymer-Based Nanoparticles for Cystic Fibrosis Lung Gene Therapy Studies Materials cystic fibrosis cystic fibrosis transmembrane conductance regulator (CFTR) lung gene delivery nanoparticles biopolymers PLGA chitosan Locked-Nucleic Acid (LNA)
author_facet	Elena Fernández Fernández Beatriz Santos-Carballal Chiara de Santi Joanne M. Ramsey Ronan MacLoughlin Sally-Ann Cryan Catherine M. Greene
author_sort	Elena Fernández Fernández
title	Biopolymer-Based Nanoparticles for Cystic Fibrosis Lung Gene Therapy Studies
title_short	Biopolymer-Based Nanoparticles for Cystic Fibrosis Lung Gene Therapy Studies
title_full	Biopolymer-Based Nanoparticles for Cystic Fibrosis Lung Gene Therapy Studies
title_fullStr	Biopolymer-Based Nanoparticles for Cystic Fibrosis Lung Gene Therapy Studies
title_full_unstemmed	Biopolymer-Based Nanoparticles for Cystic Fibrosis Lung Gene Therapy Studies
title_sort	biopolymer-based nanoparticles for cystic fibrosis lung gene therapy studies
publisher	MDPI AG
series	Materials
issn	1996-1944
publishDate	2018-01-01
description	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).
topic	cystic fibrosis cystic fibrosis transmembrane conductance regulator (CFTR) lung gene delivery nanoparticles biopolymers PLGA chitosan Locked-Nucleic Acid (LNA)
url	http://www.mdpi.com/1996-1944/11/1/122
work_keys_str_mv	AT elenafernandezfernandez biopolymerbasednanoparticlesforcysticfibrosislunggenetherapystudies AT beatrizsantoscarballal biopolymerbasednanoparticlesforcysticfibrosislunggenetherapystudies AT chiaradesanti biopolymerbasednanoparticlesforcysticfibrosislunggenetherapystudies AT joannemramsey biopolymerbasednanoparticlesforcysticfibrosislunggenetherapystudies AT ronanmacloughlin biopolymerbasednanoparticlesforcysticfibrosislunggenetherapystudies AT sallyanncryan biopolymerbasednanoparticlesforcysticfibrosislunggenetherapystudies AT catherinemgreene biopolymerbasednanoparticlesforcysticfibrosislunggenetherapystudies
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Biopolymer-Based Nanoparticles for Cystic Fibrosis Lung Gene Therapy Studies

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