New pulmonary delivery platform based on chitosan nanocapsules with potential application in gene therapy

Abstract

Gene therapy offers hope of cure for rare and other serious diseases that currently have no established effective treatments. It has become a valuable therapeutic strategy for congenital or acquired genetic lung diseases, such as alpha1-antitrypsin deficiency, infections and some types of lung cancer, among others. In this Thesis work, a new pulmonary delivery platform consisting of mannitol microspheres (Ma MS) containing chitosan-based nanocapsules (CS-based NCs) loaded with the model plasmid pCMV-betaGal is proposed for its direct delivery to the lung. The designed micro-nanostructures have suitable aerodynamic properties, are biocompatible with the A549 lung cell line and are distributed in the deep lung, producing transfection.