NVS1.1
NVS1.1 is an orally active, blood-brain barrier-penetrant eRF1 degrader. NVS1.1 induces ubiquitination of eRF1 at Lys279, mediates proteasomal degradation via the E3 ubiquitin ligases RNF14 and RNF25 as well as the translational stress sensor GCN1, traps eRF1 at the ribosomal A-site, inhibits translation termination and triggers ribosome collision. As a readthrough enhancer, NVS1.1 enables near-cognate tRNA incorporation at premature termination codons by reducing intracellular eRF1 levels. NVS1.1 activates ribosome-associated quality control pathways via ribosome collision, including ubiquitination of small subunit ribosomal proteins. NVS1.1 restores functional full-length CFTR and IDUA proteins and reduces glycosaminoglycan accumulation in relevant models. NVS1.1 can be used in the research of cystic fibrosis and Hurler syndrome (mucopolysaccharidosis type I, MPS I).
For research use only. We do not sell to patients.
- Formula: C17H16ClN3O2S
- Molecular Weight:361.85
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Storage:
Please store the product under the recommended conditions in the Certificate of Analysis.
Biological Activity
Description
IC50 & Target
[1]|
eRF1 |
RNF14 |
RNF25 |
In Vitro
NVS1.1 (1-10 μM; 48 h) dose-dependently restores functional IDUA enzymatic activity in HEKR4 cells expressing the IDUAQ70X or IDUAW402X nonsense mutations[1].
NVS1.1 (0.1-10 μM for 7 days, 0-0.6 μM for 7 weeks) dose-dependently restores IDUA activity, reduces GAG accumulation, normalizes GUSB activity, and is accompanied by eRF1 depletion in primary fibroblasts from homozygous IDUAW402X Hurler syndrome patients[1].
NVS1.1 (0.00-5.00 μM; 8-24 h) increases the level of IDUA-W402X mRNA in primary fibroblasts from Hurler syndrome patients[1].
NVS1.1 (2.5 μM; 6 h) induces proteasomal degradation of eRF1 in HEKR4 PTC reporter cells, and this degradation process does not require neddylation modification of Cullin-RING ligases[1].
NVS1.1 (25 μM; 30 min) induces ubiquitination modification of eRF1 in HeLa and HEKR4 PTC cells[1].
The process by which NVS1.1 (0.00-2.5 μM; 6 h) induces eRF1 degradation in HEKR4 PTC reporter cells requires the catalytically active E3 ubiquitin ligases RNF14 and RNF25[1].
The nonsense codon readthrough-inducing effect of NVS1.1 (0.1-10 μM; 24 h) in HEKR4 PTC reporter cells depends on catalytically active RNF14 and RNF25[1].
NVS1.1 (2.5 μM; 6 h) requires GCN1 to induce the degradation of eRF1 in HEKR4 cells[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only. Further protocols information, click here.
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Cell Line:Parental, RNF14/RNF25 knockout, and rescue HEKR4 PTC reporter cells
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Concentration:0, 0.16, 0.31, 0.63, 1.25, 2.50 μM
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Incubation Time:6 h
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Result:Decreased eRF1 levels in a dose-dependent manner in parental cells.
Rendered cells resistant to NVS1.1-induced eRF1 degradation in RNF14 or RNF25 knockout cells.
Restored NVS1.1 sensitivity in rescue cell lines expressing wild-type RNF14 or RNF25, while catalytically inactive mutants (RNF14 C220S, RNF25 C135S/C138S) did not.
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Cell Line:primary Hurler syndrome fibroblasts
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Concentration:0, 0.76, 1.95, 5
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Incubation Time:8, 16, 24 h
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Result:Did not affect IDUA-W402X mRNA levels in primary Hurler syndrome fibroblasts at concentrations ≤2 μM across 8-24 h, but increased mRNA levels at 5 μM after 16-24 h
In Vivo
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:Wistar Kyoto (homozygous for IDUA-W401X mutation; male and female; 8-10 weeks old; 200-300 g)[1]
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Dosage:20 mg/kg; 40 mg/kg
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Administration:p.o.; daily; 14 days
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Result:Restored approximately 1% of the IDUA activity measured in brain tissue of untreated wild-type rats (40 mg/kg dose).
Reduced brain GAG levels by 57% (40 mg/kg dose).
Reduced GUSB activity to approximately 1.3-fold of wild-type levels (40 mg/kg dose).
Reduced brain GAG levels by 36% (20 mg/kg dose).
Chemical Information
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Molecular Weight 361.85
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Formula C17H16ClN3O2S
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SMILES
N[C@H](C1=CC=CC=C1)C(C=C2S(=O)(C)=O)=C(C=C2C3=CC=NN3)Cl
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
Please store the product under the recommended conditions in the Certificate of Analysis.
Protocols
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Fibrosis/Collagen Morphometry
Fibrosis and collagen morphometry is based on the quantitative visualization of fibrillar collagen deposition in tissue sections using histochemical stains such as Sirius Red (Picrosirius Red) or Masson's trichrome, followed by image-based or polarization-enhanced analysis to estimate collagen proportional area as a surrogate of extracellular matrix accumulation during fibrotic remodeling. Sirius Red combined with polarized light microscopy enhances detection of collagen fibers due to birefringence properties, enabling more specific visualization of collagen type I and III fibrils compared to conventional bright-field histology, while whole-section or region-restricted digital morphometry reduces field-selection bias in fibrosis assessment. Alternative quantitative approaches include second harmonic generation (SHG) and two-photon excited fluorescence microscopy, which enable label-free detection of fibrillar collagen and have been validated against histological staining and biochemica
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Collagen: Sirius Red Staining
Sirius Red or picrosirius red staining is a histochemical method for visualizing collagen-rich extracellular matrix in tissue sections, and collagen fibers are detected as red-stained structures under bright-field microscopy with enhanced birefringence under polarized light. Picrosirius red is useful for assessing total collagen organization, distribution, and fibrosis burden, but polarized color should not be interpreted as a definitive collagen type I versus type III readout because color is affected by fiber orientation, thickness, and packing.
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Connective Tissue: Masson's Trichrome/Collagen Trichrome Staining
Masson’s Trichrome (collagen/trichrome staining) is a histological technique that differentially stains tissue compartments using sequential acidic dyes to distinguish collagen from muscle and cytoplasmic components based on dye affinity and tissue permeability differences, enabling visualization of fibrosis and connective tissue architecture in histological sections. The classical formulation typically uses Weigert's iron hematoxylin for nuclear staining, Biebrich scarlet-acid fuchsin for cytoplasm and muscle, and aniline blue (or light green variants) for collagen, producing a characteristic blue/green collagen signal contrasted against red cytoplasm and dark nuclei. The staining principle relies on selective displacement of smaller dye molecules by larger anionic dyes in collagen-rich regions under controlled acidified conditions, which enhances collagen-specific dye retention. This property makes the method widely used for fibrosis assessment in organs such as heart, liver, lung, a
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Carbohydrates and Mucins: Alcian Blue/Alcian Blue-PAS Staining
Alcian Blue (AB) staining is a cationic copper phthalocyanine dye-based histochemical method that binds electrostatically to negatively charged acidic mucopolysaccharides (glycosaminoglycans and sialomucins), enabling visualization of acidic carbohydrate-rich structures such as epithelial mucins, cartilage matrix, and mast cell granules. Periodic Acid-Schiff (PAS) reaction detects neutral mucopolysaccharides and glycoconjugates by oxidizing vicinal diols to aldehydes, which subsequently react with Schiff reagent to produce a magenta signal. The combined Alcian Blue-PAS (AB-PAS) method allows simultaneous differentiation of acidic (blue) and neutral (magenta) mucins in the same tissue section, enabling mucin subtype discrimination in epithelial tissues and pathological lesions.
Purity & Documentation
References
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)