Given the high attrition rates, substantial costs and slow pace of new drug discovery and
development, repurposing of'old'drugs to treat both common and rare diseases is …

Cystic fibrosis (CF) is the most common life-threatening monogenic disease afflicting
Caucasian people. It affects the respiratory, gastrointestinal, glandular and reproductive …

Available corrector drugs are unable to effectively rescue the folding defects of CFTR-ΔF508
(or CFTR-F508del), the most common disease-causing mutation of the cystic fibrosis …

Opioid use disorder (OUD) is a chronic and relapsing condition that involves the continued
and compulsive use of opioids despite harmful consequences. The development of …

Cystic fibrosis (CF) is caused by mutations in the gene encoding the cystic fibrosis
transmembrane conductance regulator (CFTR) that compromise its chloride channel activity …

The peripheral protein quality control (QC) system removes non-native membrane proteins,
including ΔF508-CFTR, the most common CFTR mutant in cystic fibrosis (CF), from the …

Pharmacological chaperones represent a class of therapeutic compounds for treating
protein misfolding diseases. One of the most prominent examples is the FDA-approved …

Remarkable progress in CFTR research has led to the therapeutic development of
modulators that rescue the basic defect in cystic fibrosis. There is continuous interest in …

Currently available antiepileptic drugs (AEDs) fail to control seizures in 30% of patients.
Genomics-based drug repurposing (GBR) offers the potential of savings in the time and cost …

Conformationally defective cystic fibrosis transmembrane conductance regulator (CFTR)
including rescued ΔF508-CFTR is rapidly eliminated from the plasma membrane (PM) even …