SML2448

PK4C9

≥98% (HPLC)

• 別名: Homocarbonyltopsentin, [2-[(6-Hydroxy-1H-indol-3-yl)carbonyl]-1H-imidazol-4-yl]-1H-indol-3-yl--methanone
• 実験式（ヒル表記法）: C₂₁H₁₄N₄O₃
• CAS番号: 172286-77-0
• 分子量: 370.36
• UNSPSCコード: 12352200
• NACRES: NA.77

特性

• アッセイ: ≥98% (HPLC)
• 形状: powder
• 色: white to very dark brown
• 溶解性: DMSO: 2 mg/mL, clear
• 保管温度: 2-8°C

詳細

生物化学的／生理学的作用

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.

安全性情報

• 保管分類コード: 11 - Combustible Solids
• WGK: WGK 3
• 引火点(°F): Not applicable
• 引火点(℃): Not applicable