Exaluren, also known as NB-124 and ELX 02 is a a synthetic aminoglycoside and a translational read-through inducer. ELX-02 suppresses translation termination at PTCs (i.e., readthrough) by promoting the insertion of an amino acid at the PTC and restoring expression of full-length CFTR protein. After treatment with ELX-02 and CFTR correctors, CFTR function rescued from the G550X allele in FRTs reached 20–40% of the wild-type level. Exaluren is being developed as a therapy for genetic diseases caused by nonsense mutations.
MedKoo Cat#: 574270
Name: Exaluren free base
CAS#: 1375073-93-0 (free base)
Chemical Formula: C19H38N4O10
Exact Mass: 482.2588
Molecular Weight: 482.53
Elemental Analysis: C, 47.29; H, 7.94; N, 11.61; O, 33.16
The following data is based on the product molecular weight 482.53 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.
| Concentration / Solvent Volume / Mass | 1 mg | 5 mg | 10 mg |
|---|---|---|---|
| 1 mM | 1.15 mL | 5.76 mL | 11.51 mL |
| 5 mM | 0.23 mL | 1.15 mL | 2.3 mL |
| 10 mM | 0.12 mL | 0.58 mL | 1.15 mL |
| 50 mM | 0.02 mL | 0.12 mL | 0.23 mL |
