Selective SMN2 gene splicing modifier
Risdiplam is a selective survival of motor neuron-2 (SMN2) pre-mRNA splicing modifier. It potently enhances SMN2 splicing
in vitro
(EC1.5X = 4 nM) and increases SMN protein in both brain (up to 100%) and muscle tissues (up to 49%) of transgenic mouse models of spinal muscular atrophy (SMA). Risdiplam dose-dependently increases the number of vesicular glutamate transporter 1 (vGlut1) proprioceptive inputs on motor neurons, the number of L3 to L5 motor neurons, the percentage of fully innervated neuromuscular junctions, and muscle size in the extensor digitorum longus muscles in SMN Δ7 mice.
| Marque | Tocris Bioscience |
|---|---|
| Réf. B-LABO | BL-RE-124456 |
| Réf. fabricant | 18775955 |
| Nom chimique ou matériau | 7-(4,7-Diazaspiro[2.5 ]oct-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)-4 H-pyrido[1,2-a]pyrimidin-4-one |
| CAS | 1825352-65-5 |
| Quantité | 50 mg |
| Cible | DNA, RNA and Protein Synthesis Compounds |
| Formule moléculaire | C<sub>22</sub>H<sub>23</sub>N<sub>7</sub>O |
| Pureté | 0.98 |
