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Branaplam (LMI070) HCl

Branaplam (LMI070) HCl

Item no.	V3853-500mg
Manufacturer	InvivoChem LLC
CASRN	1562338-39-9
Amount	500 mg
Quantity options	100 mg 250 mg 25 mg 50 mg
Category	Reagents & Chemicals Molecules
Type	Biochemicals
Specific against	other
ECLASS 10.1	32160490
ECLASS 11.0	32160490
UNSPSC	12000000
Available
Description	InvivoChem Cat #:V3853CAS #:1562338-39-9 Purity >=98% Description: LMI070 (also known as Branaplam and NVS-SM1) is a potent, orally active, small-molecule enhancer of SMN2 (survival of motor neuron-2) splicing that elevates full-length SMN protein and extends survival in a severe SMA mouse model. The molecular mechanism of action is via stabilization of the transient double-strand RNA structure formed by the SMN2 pre-mRNA and U1 small nuclear ribonucleic protein (snRNP) complex. The binding affinity of U1 snRNP to the 5' splice site is increased in a sequence-selective manner, discrete from constitutive recognition. This new mechanism demonstrates the feasibility of small molecule-mediated, sequence-selective splice modulation and the potential for leveraging this strategy in other splicing diseases. References: Nat Chem Biol. 2015 Jul; 11(7):511-7. Related CAS#:1562338-42-4 (free base)
Molecular Weight (MW)	429.94
Formula	C22H28ClN5O2
CAS No.	1562338-39-9 (HCl)
Storage	-20C for 3 years in powder form
	-80C for 2 years in solvent
Solubility (In vitro)	DMSO: 6 mg/mL
	Water: N/A
	Ethanol: N/A
Chemical Name	(6Z)-3-(1H-pyrazol-4-yl)-6-[3-(2, 2, 6, 6-tetramethylpiperidin-4-yl)oxy-1H-pyridazin-6-ylidene]cyclohexa-2, 4-dien-1-one hydrochloride
Synonyms	LMI070 HCl; NVS SM1 hydrochloride; LMI 070; NVS-SM1 HCl; LMI-070; NVSSM1 hydrochloride;
SMILES Code	OC1=CC(C2=CNN=C2)=CC=C1C3=NN=C(OC4CC(C)(C)NC(C)(C)C4)C=C3.[H]Cl

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Amount: 500 mg

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