Biological Activity
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).
In Vitro
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In Vivo
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References & source
1. Shiori Ando, et al. Sci Rep. 2020 Oct 15;10(1):17472.
Published experimental findings depend on the model and assay conditions. They do not guarantee equivalent performance in every application. For laboratory research use only.











