Bioactivity | Exaluren (ELX-02) is an synthetic eukaryotic ribosome-selective glycoside that induces read through of nonsense mutations, resulting in normally localized full-length functional proteins. Exaluren is used for the research of cystic fibrosis caused by nonsense mutations[1]. |
Name | Exaluren |
CAS | 1375073-93-0 |
Formula | C19H38N4O10 |
Molar Mass | 482.53 |
Transport | Room temperature in continental US; may vary elsewhere. |
Storage | Please store the product under the recommended conditions in the Certificate of Analysis. |
Reference | [1]. Leubitz A, Frydman-Marom A, Sharpe N, van Duzer J, Campbell KCM, Vanhoutte F. Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX-02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations, in Healthy Volunteers. |