Nonsense mutations promote premature translational termination and cause anywhere from
5–70% of the individual cases of most inherited diseases 1 . Studies on nonsense-mediated …

Spinal muscular atrophy (SMA) is a genetic disease caused by mutation or deletion of the
survival of motor neuron 1 (SMN1) gene. A paralogous gene in humans, SMN2, produces low, …

To examine the role of the mitochondrial polymerase (Pol γ) in clinically observed toxicity of
nucleoside analogs used to treat AIDS, we examined the kinetics of incorporation catalyzed …

Hypericin is a polycyclic anthrone first isolated from the plant St. Johnswort and was shown
to have dramatic anti-retroviral activity against Friend leukemia virus and radiation leukemia …

The thymidine analog fialuridine deoxy-2-fluoro-beta-D-arabinofuranosyl)-5-iodouracil (FIAU)
was toxic in trials for chronic hepatitis B infection. One mechanism postulated that …

Huntington’s disease (HD) is a hereditary neurodegenerative disorder caused by expansion
of cytosine-adenine-guanine (CAG) trinucleotide repeats in the huntingtin (HTT) gene. …

The coronavirus disease 2019 (COVID-19) pandemic has created an urgent need for
therapeutics that inhibit the SARS−COV-2 virus and suppress the fulminant inflammation …

The NS5B RNA-dependent RNA polymerase encoded by hepatitis C virus (HCV) plays a
key role in viral replication. Reported here is evidence that HCV NS5B polymerase acts as a …

PTC299 was identified as an inhibitor of VEGFA mRNA translation in a phenotypic screen
and evaluated in the clinic for treatment of solid tumors. To guide precision cancer treatment, …

Nonsense mutations, resulting in a premature stop codon in the open reading frame of
mRNAs are responsible for thousands of inherited diseases. Readthrough of premature stop …