| Identification | Back Directory | [Name]
INDEX NAME NOT YET ASSIGNED | [CAS]
2229964-07-0 | [Synonyms]
RG4326
RGLS4326 | [Molecular Formula]
C95H115F3N32O51P8S8 | [Molecular Weight]
3082.42 |
| Hazard Information | Back Directory | [Uses]
RGLS4326 (RG4326) is a first-in-class, short oligonucleotide inhibitor of microRNA-17 (miR-17). RGLS4326 can be used for the research of autosomal dominant polycystic kidney disease (ADPKD). RGLS4326 inhibits miR-17 function in HeLa cells with an EC50 value of 28.3?nM[1]. | [in vivo]
RGLS4326 preferentially distributes to kidney tubules and cysts. RGLS4326 (a single 30?mg/kg SC injection) is rapidly absorbed into plasma, showing Tmax of ≤1?h, Cmax of 8.5?μg/mL, and half-life of <4?h in wild-type mice[1].
In vivo administration of RGLS4326 also upregulates the expression of the direct miR-17 target genes Pkd1 and Pkd2[1]. | Animal Model: | Pkd2-KO mice[1] | | Dosage: | 20?mg/kg | | Administration: | SC injection | | Result: | Compared to non-cystic control kidneys, polycystic kidneys of PBS-treated Pkd2-KO mice exhibit an age-dependent progressive decline in miR-17 PD-Sig, indicative of increasing miR-17 activity with disease progression.
Administration of RGLS4326 reversed this decline in miR-17 PD-Sig, indicating a sustained functional inhibition of miR-17. |
| [References]
[1] Edmund C Lee, et al. Discovery and preclinical evaluation of anti-miR-17 oligonucleotide RGLS4326 for the treatment of polycystic kidney disease. Nat Commun.?2019 Sep 12;10(1):4148. DOI:10.1038/s41467-019-11918-y |
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