An SMN2 splice modulator; enhances SMN2 splicing activity by stabilizing the snRNP-SMN2 pre-mRNA complex; increases the expression of the full-length SMN2 transcript at 3-20 mg/kg; increases SMN protein levels in the brain and spinal cord in the C/+ mouse model of SMA at 0.3-30 mg/kg; increases survival of SMNΔ7 mice at 1 and 3 mg/kg

LMI070 – 1 mg

Brand:
Cayman
CAS:
1562338-42-4
Storage:
-20
UN-No:
Non-Hazardous - /

LMI070 is an SMN2 splice modulator.{42746} It selectively enhances SMN2 splicing by stabilizing the complex formed by the U1 small nuclear ribonucleic protein (snRNP) and SMN2 pre-mRNA. LMI070 (3-30 mg/kg) increases the expression of the full-length SMN2 trancript and, at doses ranging from 0.3 to 30 mg/kg, increases SMN protein levels in the brain and spinal cord in the C/+ mouse model of spinal muscular atrophy (SMA).{42746,42747} It also increases survival of SMNΔ7 mice, a model of severe SMA, when administered at doses of 1 and 3 mg/kg.{42746}  

 

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Description

An SMN2 splice modulator; enhances SMN2 splicing activity by stabilizing the snRNP-SMN2 pre-mRNA complex; increases the expression of the full-length SMN2 transcript at 3-20 mg/kg; increases SMN protein levels in the brain and spinal cord in the C/+ mouse model of SMA at 0.3-30 mg/kg; increases survival of SMNΔ7 mice at 1 and 3 mg/kg

Formal name: 5-(1H-pyrazol-4-yl)-2-[6-[(2,2,6,6-tetramethyl-4-piperidinyl)oxy]-3-pyridazinyl]-phenol

Synonyms:  Branaplam|NVS-SM1

Molecular weight: 393.5

CAS: 1562338-42-4

Purity: ≥98%

Formulation: A crystalline solid

Product Type|Biochemicals||Research Area|Neuroscience

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