Air− liquid interface (ALI) culture models currently represent a valid instrument to recreate
the typical aspects of the respiratory tract in vitro in both healthy and diseased state. They …

The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible
progress in treating the disease such that the mean survival age of individuals living with CF …

Ionizable lipids are a key component of lipid nanoparticles, the leading nonviral messenger
RNA delivery technology. Here, to advance the identification of ionizable lipids beyond …

Inhaled siRNA therapy has a unique potential for treatment of severe lung diseases, such as
cystic fibrosis (CF). Nevertheless, a drug delivery system tackling lung barriers is mandatory …

Over the past 30 years, a range of cystic fibrosis (CF) animal models have been generated
for research purposes. Different species, including mice, rats, ferrets, rabbits, pigs, sheep …

Cystic fibrosis (CF) is a multiorgan recessive genetic disease caused by mutations in the
cystic fibrosis transmembrane conductance regulator (CFTR) gene. Gene therapy efforts …

Rationale: Classical interpretation of cystic fibrosis (CF) lung disease pathogenesis
suggests that infection initiates disease progression, leading to an exuberant inflammatory …

Developing safe and efficient non-viral delivery systems remains a major challenge for in
vivo applications of gene therapy, especially in cystic fibrosis. Unlike conventional cationic …

Since the early days of its conceptualization and application, human gene transfer held the
promise of a permanent solution to genetic diseases including cystic fibrosis (CF). This field …

Highly-effective cystic fibrosis transmembrane conductance regulator (CFTR) modulator
therapy (HEMT) corrects the underlying molecular defect causing cystic fibrosis (CF) …