Catalog No.: PC-21294Not For Human Use, Lab Use Only.
NVS1.1 is a potent readthrough promoter, promotes the rapid and specific degradation of eukaryotic release factor 1 (eRF1) and restores functional full-length proteins in cystic fibrosis and Hurler disease models.
NVS1.1 is a potent readthrough promoter, promotes the rapid and specific degradation of eukaryotic release factor 1 (eRF1) and restores functional full-length proteins in cystic fibrosis and Hurler disease models.
NVS1.1 restores α-L-iduronidase (IDUA) enzyme activity and reduces the GAG levels in a dose-dependent manner in human patient-derived fibroblasts.
NVS1.1 stimulates premature termination codon (PTC) suppression by triggering rapid proteasomal degradation of the translation termination factor eRF1.
NVS1.1 blocks translation termination by trapping eRF1 in the A site, causing ribosome collisions.
NVS1.1 is efficacious in a rat Hurler IDUA-W401X animal model, shows good drug-like properties with favorable pharmacokinetics, metabolic stability, and suitability for oral administration.
