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).
Keywords: Locked-Nucleic Acid (LNA); PLGA; biopolymers; chitosan; cystic fibrosis; cystic fibrosis transmembrane conductance regulator (CFTR); lung gene delivery; nanoparticles.