| Cas No.: | 2407723-70-8 |
| Chemical Name: | SMN2 splicing modulator TEC-1 |
| Formula: | C21H23N7O |
| M.Wt: | 389.463 |
| Purity: | >98% |
| Sotrage: | 2 years -20°C Powder, 2 weeks 4°C in DMSO, 6 months -80°C in DMSO |
| Publication: | 1. Shiori Ando, et al. Sci Rep. 2020 Oct 15;10(1):17472. |
| Description: | SMN2 splicing modulator TEC-1 is a novel specific, CNS penetrant small molecule SMN2 splicing modulator, increases the expression level of FL-SMN2 mRNA and decreases the expression level of Δ7 mRNA.TEC-1 showed higher selectively (>60-fold) on galactosylceramidase and huntingtin gene expression compared to previously reported compounds (e.g., SMN-C3) due to off-target effects on cryptic exon inclusion and nonsense-mediated mRNA decay.TEC-1 modulates SMN2 splicing and displays disease-modifying effects in motor neurons derived from SMA patient iPSCs (GM24468).|TEC-1 rescues the phenotype in a murine model of spinal muscular atrophy (SMA). |
| References: | 1. Shiori Ando, et al. Sci Rep. 2020 Oct 15;10(1):17472. |

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