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

  1. Estefanía Fernández Paz
Dirixida por:
  1. Carmen Remuñán López Director

Universidade de defensa: Universidade de Santiago de Compostela

Ano de defensa: 2022

  1. Departamento de Farmacoloxía, Farmacia e Tecnoloxía Farmacéutica

Tipo: Tese


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.