ML372 inhibits survival motor neuron (SMN) protein ubiquitination, increases SMN protein stability without affecting mRNA expression. ML372 improves spinal muscular atrophy (SMA) in mice. ML372 is brain penetrant and has a reasonable exposure and half-life in vivo.
性状
Solid
运输条件
Room temperature in continental US; may vary elsewhere.
储存方式
Powder -20°C 3 years;4°C 2 years
参考文献
[1]. Mahlet B Abera, et al. ML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in mice. JCI Insight. 2016 Nov 17;1(19):e88427.
溶解度数据
In Vitro: DMSO : 33.33 mg/mL (92.47 mM; ultrasonic and warming and heat to 60°C)配制储备液
[1]. Mahlet B Abera, et al. ML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in mice. JCI Insight. 2016 Nov 17;1(19):e88427.