For complex diseases, most drugs are highly ineffective, and the success rate of drug
discovery is in constant decline. While low quality, reproducibility issues, and translational …

Following the discovery of nucleic acids by Friedrich Miescher in 1868, DNA and RNA were
recognized as the genetic code containing the necessary information for proper cell …

Importance Cystic fibrosis, a genetic disorder defined by variants in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene, affects more than 30 000 individuals in …

Human diseases have been a critical threat from the beginning of human history. Knowing
the origin, course of action and treatment of any disease state is essential. A microscopic …

Respiratory cilia are the driving force of the mucociliary escalator, working in conjunction
with secreted airway mucus to clear inhaled debris and pathogens from the conducting …

Gene therapy for neurological disorders has attracted significant interest as a way to reverse
or stop various disease pathologies. Typical gene therapies involving the central and …

Cystic fibrosis (CF) is a life-threatening autosomal-recessive disease caused by mutations in
a single gene encoding cystic fibrosis transmembrane conductance regulator (CFTR). CF …

Mutations in the CFTR gene that lead to premature stop codons or splicing defects cause
cystic fibrosis (CF) and are not amenable to treatment by small-molecule modulators. Here …

Gene therapy encompasses the transfer of exogenous genetic materials into the patient's
target cells to treat or prevent diseases. Nevertheless, the transfer of genetic material into …

Respiratory disorders are among the most important medical problems threatening human
life. The conventional therapeutics for respiratory disorders are hindered by insufficient drug …