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Chemical Structure : NVS1.1
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.
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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.
| M.Wt | 361.84 | |
| Formula | C17H16ClN3O2S | |
| Appearance | Solid | |
| Storage |
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| Solubility |
10 mM in DMSO |
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1. Gurzeler LA, et al. Cell Rep. 2023 Sep 26;42(9):113056.
2. Patent WO2014/091446A1
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