For research use only. Not for therapeutic Use.
PTC124 (Cat No.: I004853) is an investigational small molecule designed to address genetic disorders caused by nonsense mutations, such as Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF). It promotes ribosomal readthrough of premature stop codons, enabling the production of functional proteins. PTC-124 specifically targets nonsense mutations without affecting normal termination codons, offering a precise therapeutic approach. Widely studied in clinical trials, it holds promise for treating various genetic diseases by restoring partial functionality to defective proteins, improving patient outcomes.
| Catalog Number | I004853 |
| CAS Number | 775304-57-9 |
| Synonyms | 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid |
| Molecular Formula | C₁₅H₉FN₂O₃ |
| Purity | ≥95% |
| Target | Autophagy |
| Solubility | DMSO: ≥ 52 mg/mL |
| Storage | store at -20℃ |
| IUPAC Name | 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid |
| InChI | InChI=1S/C15H9FN2O3/c16-12-7-2-1-6-11(12)14-17-13(18-21-14)9-4-3-5-10(8-9)15(19)20/h1-8H,(H,19,20) |
| InChIKey | OOUGLTULBSNHNF-UHFFFAOYSA-N |
| SMILES | C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F |
