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SMN-C3
本产品不向个人销售,仅用作科学研究,不用于任何人体实验及非科研性质的动物实验。
SMN-C3图片
包装与价格:
包装价格(元)
250mg电议
500mg电议

产品介绍
SMN-C3是一种可口服的SMN2剪接调节剂,有治疗脊椎肌肉萎缩(SMA)的潜力。

Animal experiment:

Mice[1]The animals are treated with SMN-C3 at doses of 0.3, 1, and 3 mg/kg per day by intraperitoneal injections from P3 through P23 and thereafter at doses of 1, 3, and 10 mg/kg per day, respectively, by oral gavage[1].

产品描述

SMN-C3 is an orally active SMN2 splicing modulator and has the potential to treat spinal muscular atrophy (SMA).

At P16, vehicle treated D7 mice are much smaller than heterozygous littermate controls and appear moribund. In contrast, D7 mice treated with the high dose of SMN-C3 show a phenotype similar to that of heterozygous controls. SMN-C3 treatment induces a dose-dependent bodyweight gain in the D7 mice, with some animals showing a body weight that is ~80% that of heterozygous controls. SMN-C3 normalizes the motor behavior of D7 mice, illustrated by the ability of the mice to right themselves as quickly as heterozygous controls and by their level of locomotor activity. Most importantly, whereas vehicle-treated mice die within 3 weeks after birth with a median survival of 18 days, SMN-C3 treatment increases survival in a dose-dependent manner to a median survival time of 28 days in the low-dose (0.3 mg/kg per day) group. In the two higher-dose groups (1 and 3 mg/kg per day), ~90% of animals survive beyond P65 when the study is completed[1].

[1]. Naryshkin NA, et al. Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy. Science. 2014 Aug 8;345(6197):688-93.