NVS1.1 ammonium acetate

NVS1.1 (1-10 μM; 48 h) dose-dependently restores functional IDUA enzymatic activity in HEKR4 cells expressing the IDUA^Q70X or IDUA^W402X 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 IDUA^W402X 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].