Albert Iron9, Herve´ Mittre10, Delphine Feldmann11, Caroline Guittard3, Mireille Claustres1,2,3 and Marie des Georges3 Among the 1700 mutations reported in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, a missense mutation, p.Ser1235Arg, is a relatively frequent finding....
Cystic Fibrosis (CF; OMIM: no 210700) is an autosomal recessive genetic disorder resulting from pathogenic variants in the CFTR (Cystic Fibrosis Transmembrane Regulator; at position 7q3.1) gene, which mainly affects White patients [1]. In Brazil, its estimated prevalence is 1:7500–15,000, var...
Keywords: cystic fibrosis; cystic fibrosis transmembrane conductance regulator (CFTR); lung gene delivery; nanoparticles; biopolymers; PLGA; chitosan; Locked-Nucleic Acid (LNA) 1. Introduction The most frequent lethal genetic disease in Caucasian populations is Cystic Fibrosis (CF). Mutations in the ...