Risdiplam RG7916; RO7034067,99.35%
产品编号:Bellancom-109101| CAS NO:1825352-65-5| 分子式:C22H23N7O| 分子量:401.46
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Risdiplam RG7916; RO7034067
产品介绍 | Risdiplam (RG7916) 是一种运动神经元存活基因 SMN2 的前体 mRNA 剪接修饰剂,可增加 SMN 蛋白水平。 | ||||||||||||||||
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生物活性 | Risdiplam (RG7916) is an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier that increases survival motor neuron (SMN) protein levels. | ||||||||||||||||
体外研究 |
Risdiplam modulates SMN2 pre-mRNA splicing towards the production of full-length SMN2 mRNA and increases SMN protein levels. Risdiplam is a modifier of SMN2 splicing, leading to an increase in SMN2 full length transcript and thus functional SMN protein. Spinal muscular atrophy (SMA) type I remains the most common genetic disease resulting in death in infancy. Characterized by progressive motor and respiratory muscle weakness, this autosomal recessive neuromuscular disorder is caused by low levels of the survival motor neuron protein (SMN) due to inactivating bi-allelic deletions and other disabling mutations in the survival motor neuron 1 (SMN1) gene. 西域 has not independently confirmed the accuracy of these methods. They are for reference only. | ||||||||||||||||
体内研究 | |||||||||||||||||
体内研究 | |||||||||||||||||
性状 | Solid | ||||||||||||||||
溶解性数据 |
In Vitro:
DMSO : 1.7 mg/mL (4.23 mM; ultrasonic and warming and heat to 60°C) Ethanol : < 1 mg/mL (insoluble) H2O : < 0.1 mg/mL (insoluble) *Risdiplam is usually formulated as a suspension. 配制储备液
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请根据产品在不同溶剂中的溶解度选择合适的溶剂配制储备液;一旦配成溶液,请分装保存,避免反复冻融造成的产品失效。 | ||||||||||||||||
运输条件 | Room temperature in continental US; may vary elsewhere. | ||||||||||||||||
储存方式 |
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参考文献 |