产品
编 号：F753981
分子量：6530.3
分子量：6530.3
产品类型
规格
价格
是否有货
1mg
询价
询价
5mg
询价
询价
10mg
询价
询价
结构图
CAS No: 2591587-57-2
产品详情
生物活性：
Rugonersen (RG6091; RO7248824) is a locked-nucleic acid (LNA)- modified antisense oligonucleotides (ASOs), and results in reduction of ubiquitin-protein ligase E3A (UBE3A) silencing. Angelman syndrome (AS) is a severe neurodevelopmental disorder caused by the loss of neuronal E3 ligase UBE3A, Rugonersen has been used for AS reasearch.
体内研究：
Rugonersen (RO7248824) (24 mg/monkey; i.t.; for 8-85 d) is well tolerated without adverse in-life effects or tissue pathology and produced a robust, long lasting (up to 3 months) paternal reactivation of UBE3A mRNA/protein across key monkey brain regions.Male cynomolgus monkeysRugonersen (150 μg; i.c.v.; single dose) selectively and potently reduces UBE3A-ATS, while concomitantly upregulating the UBE3A mRNA and protein.Animal Model:Male cynomolgus monkey
Dosage:24 mg per monkey
Administration:Intrathecal injection; single dose or twice dose with 2 weeks apart; sacrificed at 8, 15, 29, 57, and 85 days after the last dose
Result:Resulted a long duration of action on paternal UBE3A reactivation in NHP brains after IT delivery.
Animal Model:WT and AS Ube3a m-/p+ mice adult mice (10-12 weeks old)
Dosage:150 μg per mice
Administration:Intracerebroventricular injection; single dose; harvested at 2 weeks post injection
Result:Revealed a steep relationship between UBE3A-ATS knock-down and UBE3A mRNA/protein upregulation, whereby an almost 90% downregulation was needed to achieve a 50% upregulation, respectively.
体外研究：
RO7248824 (0-10 μM) show a nanomolar potency against UBE3A-ATS (EC50=26.3 nM), UBE3A mRNA upregulation (EC50=15.4 nM) and UBE3A protein upregulation (EC50=24.8 nM) in Angelman syndrome (AS) neurons.
Rugonersen (RG6091; RO7248824) is a locked-nucleic acid (LNA)- modified antisense oligonucleotides (ASOs), and results in reduction of ubiquitin-protein ligase E3A (UBE3A) silencing. Angelman syndrome (AS) is a severe neurodevelopmental disorder caused by the loss of neuronal E3 ligase UBE3A, Rugonersen has been used for AS reasearch.
体内研究：
Rugonersen (RO7248824) (24 mg/monkey; i.t.; for 8-85 d) is well tolerated without adverse in-life effects or tissue pathology and produced a robust, long lasting (up to 3 months) paternal reactivation of UBE3A mRNA/protein across key monkey brain regions.Male cynomolgus monkeysRugonersen (150 μg; i.c.v.; single dose) selectively and potently reduces UBE3A-ATS, while concomitantly upregulating the UBE3A mRNA and protein.Animal Model:Male cynomolgus monkey
Dosage:24 mg per monkey
Administration:Intrathecal injection; single dose or twice dose with 2 weeks apart; sacrificed at 8, 15, 29, 57, and 85 days after the last dose
Result:Resulted a long duration of action on paternal UBE3A reactivation in NHP brains after IT delivery.
Animal Model:WT and AS Ube3a m-/p+ mice adult mice (10-12 weeks old)
Dosage:150 μg per mice
Administration:Intracerebroventricular injection; single dose; harvested at 2 weeks post injection
Result:Revealed a steep relationship between UBE3A-ATS knock-down and UBE3A mRNA/protein upregulation, whereby an almost 90% downregulation was needed to achieve a 50% upregulation, respectively.
体外研究：
RO7248824 (0-10 μM) show a nanomolar potency against UBE3A-ATS (EC50=26.3 nM), UBE3A mRNA upregulation (EC50=15.4 nM) and UBE3A protein upregulation (EC50=24.8 nM) in Angelman syndrome (AS) neurons.
产品资料
Copyright©2015-2024 沪ICP备2022026524号-1
沪公网安备