2407723-70-8
2407723-70-8 structure
- Name: SMN2 splicing modulator TEC-1
- Chemical Name: SMN2 splicing modulator TEC-1
- CAS Number: 2407723-70-8
- Molecular Formula: C21H23N7O
- Molecular Weight: 389.463
- Create Date: 2022-07-01 17:41:51
- Modify Date: 2024-02-04 10:44:15
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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).
| Name | SMN2 splicing modulator TEC-1 |
|---|
| 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. |
| Molecular Formula | C21H23N7O |
|---|---|
| Molecular Weight | 389.463 |