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

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 cance...

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Detalhes bibliográficos
Autor: Fernández Paz, Estefanía
Formato: tesis doctoral
Fecha de publicación:2022
País:España
Recursos:Universidad de Santiago de Compostela (USC)
Repositorio:Minerva. Repositorio Institucional de la Universidad de Santiago de Compostela
Idioma:inglés
OAI Identifier:oai:minerva.usc.gal:10347/28134
Acesso em linha:http://hdl.handle.net/10347/28134
Access Level:acceso abierto
Palavra-chave:Materias::Investigación::32 Ciencias médicas::3209 Farmacología::320908 Preparación de medicamentos
Descrição
Resumo: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.