2229964-07-0
Chemical Structure
RGLS4326
Synonym(s): RG4326
- CAS No.: 2229964-07-0
- Formula:C95H115F3N32O51P8S8
- Molecular Weight:3082.42
InChIKey: WAVSYQSKUSIMAY-IOHKGNKKSA-N
SMILES: OC[C@@]1(O[C@@H](N2C3=NC=NC(N)=C3N=C2)[C@]4([H])O[C@H]1C)[C@H]4OP(OC[C@]56[C@H]([C@](O[C@H]6C)([H])[C@H](N7C8=C(C(N=C(N8)N)=O)N=C7)O5)OP(OC[C@@H]9[C@H]([C@H]([C@H](N%10C(N=C(C=C%10)N)=O)O9)OC)OP(OC[C@@H]%11[C@H]([C@H]([C@H](N%12C%13=NC=NC(N)=C%13N=C%12)O%11)F)OP(OC[C@@H]%14[C@H]([C@H]([C@H](N%15C(N=C(C=C%15)N)=O)O%14)F)OP(OC[C@@H]%16[C@H]([C@H]([C@H](N%17C(NC(C=C%17)=O)=O)O%16)F)OP(OC[C@@H]%18[C@H]([C@H]([C@H](N%19C(NC(C=C%19)=O)=O)O%18)OC)OP(OC[C@]%20%21[C@H]([C@](O[C@H]%21C)([H])[C@H](N%22C(NC(C=C%22)=O)=O)O%20)OP(OC[C@@]%23%24O[C@@H](N%25C%26=C(C(N=C(N%26)N)=O)N=C%25)[C@@](O[C@H]%24C)([H])[C@@H]%23O)(S)=O)(S)=O)(S)=O)(S)=O)(S)=O)(S)=O)(S)=O)(S)=O
Biological Activity: RGLS4326 (RG4326) is a microRNA-17 (miR-17) oligonucleotide inhibitor with an EC50 of 28.3 nM. RGLS4326 antagonizes AMPAR and inhibits ligand binding. RGLS4326 relieves the inhibition of Pkd1/Pkd2 by miR-17 and upregulates PC1/PC2. RGLS4326 preferentially distributes to the kidney and cysts, attenuates cyst progression and improves survival in mouse models of autosomal dominant polycystic kidney disease (ADPKD) in a miR-17 binding motif-dependent manner. RGLS4326 can be used in ADPKD-related research[1][2][3].
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RGLS4326 | 99.06% | RGLS4326 (RG4326) is a microRNA-17 (miR-17) oligonucleotide inhibitor with an EC50 of 28.3 nM. RGLS4326 antagonizes AMPAR and inhibits ligand binding. RGLS4326 relieves the inhibition of Pkd1/Pkd2 by miR-17 and upregulates PC1/PC2. RGLS4326 preferentially distributes to the kidney and cysts, attenuates cyst progression and improves survival in mouse models of autosomal dominant polycystic kidney disease (ADPKD) in a miR-17 binding motif-dependent manner. RGLS4326 can be used in ADPKD-related research. | ||||||||||||||||||||
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References
- [1]. Valencia T, et al. The nucleobase guanine at the 3'-terminus of oligonucleotide RGLS4326 drives off-target AMPAR inhibition and CNS toxicity. Nature communications. 2025 Nov 28;16(1):10762. [Content Brief]
- [2]. Lakhia R, et al. PKD1 and PKD2 mRNA cis-inhibition drives polycystic kidney disease progression. Nature communications. 2022 Aug 15;13(1):4765.
- [3]. Lee EC, et al. Discovery and preclinical evaluation of anti-miR-17 oligonucleotide RGLS4326 for the treatment of polycystic kidney disease. Nature communications. 2019 Sep 12;10(1):4148. [Content Brief]