Backbone cationized highly branched poly(β-amino ester)s as enhanced delivery vectors in non-viral gene therapy.

Gene therapy holds great potential for treating Lung Cystic Fibrosis (CF) which is a fatal hereditary condition arising from mutations in the CF transmembrane conductance regulator (CFTR) gene, resulting in dysfunctional CFTR protein. However, the advancement and clinical application of CF gene therapy systems have been hindered due to the absence of a highly efficient delivery vector. In this work, we introduce a new generation of highly branched poly(β-amino ester) (HPAE) gene delivery vectors for CF treatment.