SML2448

PK4C9

≥98% (HPLC)

• 别名: Homocarbonyltopsentin, [2-[(6-Hydroxy-1H-indol-3-yl)carbonyl]-1H-imidazol-4-yl]-1H-indol-3-yl--methanone
• 经验公式（希尔记法）: C₂₁H₁₄N₄O₃
• CAS号: 172286-77-0
• 分子量: 370.36
• UNSPSC代码: 12352200
• NACRES: NA.77

属性

• 方案: ≥98% (HPLC)
• 表单: powder
• 颜色: white to very dark brown
• 溶解性: DMSO: 2 mg/mL, clear
• 储存温度: 2-8°C
• SMILES字符串: [nH]1c(nc(c1)C(=O)c4c5c([nH]c4)cccc5)C(=O)c2c3c([nH]c2)cc(cc3)O
• InChI: 1S/C21H14N4O3/c26-11-5-6-13-15(9-23-17(13)7-11)20(28)21-24-10-18(25-21)19(27)14-8-22-16-4-2-1-3-12(14)16/h1-10,22-23,26H,(H,24,25)
• InChI key: DRCVQVIMGSWRLN-UHFFFAOYSA-N

说明

生化/生理作用

PK4C9 is a splice modulator of survival of motor neuron gene SMN2 that increases production of full-length SMN protein. Spinal muscular atrophy (SMA) is a motor neuron disease caused by deficiency in SMN protein resulting from loss of expression of the SMN1 gene. The related SMN2 gene can compensate, but polymorphism in SMN2 often results in altered splicing and exclusion of exon 7, which is required for a full-length SMN transcript. PK4C9 binds to pentaloop conformations of the stem-loop RNA structure TSL2, a cis-regulatory element for E7 inclusion, and promotes a shift to triloop conformations that display enhanced E7 splicing. In SMA cells, PK4C9 increased E7 inclusion by 40% accompanied by a 1.5-fold increase in SMN protein, a level shown to reverse SMA phenotypes in mice models.

Splice modulator of survival of motor neuron gene SMN2 that increases production of full-length SMN protein.

安全信息

• 储存分类代码: 11 - Combustible Solids
• WGK: WGK 3
• 闪点(°F): Not applicable
• 闪点(°C): Not applicable