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CAS No.: 1449598-06-4

RG7800 Catalog No. CSN26441

Synonyms: RO6885247

RG7800 HCl is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; has the potential to treat spinal muscular atrophy.

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生物活性

实验方案

技术信息

生物活性

描述Spinal muscular atrophy (SMA) is a common genetic cause of infant and toddler mortality. It manifests by insufficient generation of full-length survival motor neuron (SMN) protein by the SMN2 gene. RG7800 is a small molecule SMN2 splicing modifier. It promoted the inclusion of exon 7 in SMN2 mRNA to generate full-length mRNA in fibroblasts derived from a SAM type 1 patient. Oral administration of adult C/C-allele SMA mice with RG7800 (1, 3, and 10mg/kg) once daily for 10 days dose-dependently increased the level of SMN protein. In ∆7 SMA mice, intraperitoneal (i.p.) injection of RG7800 once daily for 7 days starting on postnatal day 3 (P3) resulted in a dose-dependent increase in the level of SMN protein in the brain and peripheral tissues. Also in ∆7 SMA mice, RG7800 treatment at 0.3, 1, and 3mg/kg/day (i.p.) from P3 to P23, and thereafter at 1, 3, and 10mg/kg/day, respectively, by oral gavage, resulted in a dose-dependent increase in the survival compared to the vehicle-treated group. The treatment of adult C/C-allele SMA mice with RG7800 (3 or 10mg/kg/day, p.o.) for 10 days dose-dependently corrected SMN2 splicing by including exon 7 to create full-length mRNA^[1].
参考文献

[1] Aslesh, T., Yokota, T. Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy. Cells. 2022; 11(3): 417.

实验方案

计算器摩尔计算器总药量计算器工作液计算器
储存液制备
- 1 mg
- 5 mg
- 10 mg
- 1 mM
- 2.4009 mL
- 12.0043 mL
- 24.0085 mL
- 5 mM
- 0.4802 mL
- 2.4009 mL
- 4.8017 mL
- 10 mM
- 0.2401 mL
- 1.2004 mL
- 2.4009 mL

技术信息

CAS号

1449598-06-4

储存条件

粉末: Inert atmosphere,Room Temperature

液体: -20°C:3-6 月 -80°C:12月

分子式

C24H28N6O

运输

蓝冰

分子量

416.52

别名

RO6885247

溶解度

DMSO	1.0 mg/mL (2.4 mM) warming
Water	Insoluble
Ethanol	2.0 mg/mL (4.8 mM) warming

动物实验配方

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