SMN2 splicing modulator TEC-1
Modify Date: 2024-02-04 10:44:15
SMN2 splicing modulator TEC-1 structure
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Common Name | SMN2 splicing modulator TEC-1 | ||
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| CAS Number | 2407723-70-8 | Molecular Weight | 389.463 | |
| Density | N/A | Boiling Point | N/A | |
| Molecular Formula | C21H23N7O | Melting Point | N/A | |
| MSDS | N/A | Flash Point | N/A | |
Use of SMN2 splicing modulator TEC-1SMN2 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 |
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| 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). |
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| References | 1. Shiori Ando, et al. Sci Rep. 2020 Oct 15;10(1):17472. |
| Molecular Formula | C21H23N7O |
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| Molecular Weight | 389.463 |