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Chemical Structure : SMN2 splicing modulator TEC-1
Catalog No.: PC-72088Not For Human Use, Lab Use Only.
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.
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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).
| M.Wt | 389.463 | |
| Formula | C21H23N7O | |
| Appearance | Solid | |
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1. Shiori Ando, et al. Sci Rep. 2020 Oct 15;10(1):17472.
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