Biopolymer-Based Nanoparticles for Cystic Fibrosis Lung Gene Therapy Studies

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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Bibliographic Details
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
Description
Summary: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).
ISSN:1996-1944

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