For research use only. Not for therapeutic Use.
RG3039 (Cat.No:I009026), also known as PF-06687859, is a potent DcpS inhibitor. DcpS is a therapeutic target for spinal muscular atrophy. Spinal muscular atrophy (SMA) is caused by deletion or mutation of both copies of the SMN1 gene which produces an essential protein known as SMN. RG3039 improves motor function in SMA mice. RG3039 also showed activity to improve survival, function and motor unit pathologies in two SMA mouse models.
| Catalog Number | I009026 |
| CAS Number | 1005504-62-0 |
| Synonyms | ;5-((1-(2,6-dichlorobenzyl)piperidin-4-yl)methoxy)quinazoline-2,4-diamine |
| Molecular Formula | C21H23Cl2N5O |
| Purity | ≥95% |
| Target | DcpS inhibitor |
| Solubility | Soluble in DMSO, not in water |
| Storage | 0 - 4 °C for short term, or -20 °C for long term |
| InChI | InChI=1S/C21H23Cl2N5O.2ClH/c22-15-3-1-4-16(23)14(15)11-28-9-7-13(8-10-28)12-29-18-6-2-5-17-19(18)20(24)27-21(25)26-17;;/h1-6,13H,7-12H2,(H4,24,25,26,27);2*1H |
| InChIKey | SRIYKPCSVUEGET-UHFFFAOYSA-N |
| SMILES | NC1=NC(N)=C2C(OCC3CCN(CC4=C(Cl)C=CC=C4Cl)CC3)=CC=CC2=N1.[H]Cl.[H]Cl |
| Reference | </br>1:The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse models. Gogliotti RG, Cardona H, Singh J, Bail S, Emery C, Kuntz N, Jorgensen M, Durens M, Xia B, Barlow C, Heier CR, Plasterer HL, Jacques V, Kiledjian M, Jarecki J, Rusche J, DiDonato CJ.Hum Mol Genet. 2013 Oct 15;22(20):4084-101. doi: 10.1093/hmg/ddt258. Epub 2013 Jun 4. PMID: 23736298 Free PMC Article</br>2:The DcpS inhibitor RG3039 improves motor function in SMA mice. Van Meerbeke JP, Gibbs RM, Plasterer HL, Miao W, Feng Z, Lin MY, Rucki AA, Wee CD, Xia B, Sharma S, Jacques V, Li DK, Pellizzoni L, Rusche JR, Ko CP, Sumner CJ.Hum Mol Genet. 2013 Oct 15;22(20):4074-83. doi: 10.1093/hmg/ddt257. Epub 2013 May 31. PMID: 23727836 Free PMC Article |
