Cystic fibrosis (CF) is the most common monogenic disorder, caused by mutations in the CF
transmembrane conductance regulator (CFTR) gene. Over the last 30 years, tremendous …

Cystic fibrosis (CF) is a lethal autosomal recessive genetic disease, caused by a mutation in
the cystic fibrosis transmembrane conductance regulator gene (CFTR), which primarily …

Premature termination codons (PTCs) in an mRNA ORF inactivate gene function by causing
production of a truncated protein and destabilization of the mRNA. Readthrough of a PTC …

Chronic infection and inflammation are the primary causes of declining lung function in
Cystic Fibrosis (CF) patients. ORKAMBI®(Lumacaftor-Ivacaftor) is an approved combination …

The advent of small molecule modulators targeting the cystic fibrosis transmembrane
conductance regulator (CFTR) has revolutionized the treatment of persons with cystic …

Cystic fibrosis (CF), a life-threatening multiorgan genetic disease, is facing a new era of
research and development using innovative gene-directed personalized therapies. The …

Stop codon readthrough—the decoding of a stop codon by a near-cognate tRNA—is
employed by viruses to balance levels of enzymatic and structural proteins and by …

Aminoglycosides (AG) are oligosaccharide antibiotics that interfere with the small ribosomal
subunit in aerobic, Gram-negative bacteria, causing pathogen-destructing error rates in their …

Personalized drug therapy for cystic fibrosis (CF) is a long-term dream for CF patients,
caregivers, physicians and researchers. After years of study, the fiction of personalized …

Nonsense mutations generate in-frame stop codons in mRNA leading to a premature arrest
of translation. Functional consequences of premature termination codons (PTCs) include the …