1971086-99-3
Chemical Structure
BPN-15477
- CAS No.: 1971086-99-3
- Formula:C12H10ClN5
- Molecular Weight:259.69
IUPAC Name: methyl (S)-6-methyl-5,6,7,8-tetrahydroimidazo[1,5-a]pyrazine-1-carboxylate
InChIKey: JAAXPOGNLKNTBW-UHFFFAOYSA-N
SMILES: ClC1=NC2=C(C=CN2)C(NCC3=CC=NC=C3)=N1
Biological Activity: BPN-15477 is an orally active, blood-brain barrier-permeable splicing modulator compound (SMC). BPN-15477 restores the correct splicing of exon 20 of the elongator complex protein 1 (ELP1) (EC50 = 1.9 μM), thereby significantly increasing the in vivo functional protein levels in all tissues including the brain. BPN-15477 can be used in the research of familial dysautonomia, such as frontotemporal dementia[1][2].
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BPN-15477 | 99.92% | BPN-15477 is an orally active, blood-brain barrier-permeable splicing modulator compound (SMC). BPN-15477 restores the correct splicing of exon 20 of the elongator complex protein 1 (ELP1) (EC50 = 1.9 μM), thereby significantly increasing the in vivo functional protein levels in all tissues including the brain. BPN-15477 can be used in the research of familial dysautonomia, such as frontotemporal dementia. | ||||||||||||||||||||
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[1]. Gao D, et al. A deep learning approach to identify gene targets of a therapeutic for human splicing disorders. Nat Commun. 2021 Jun 7;12(1):3332.
[Content Brief] -
[2]. Chekuri A, et al. Selective retinal ganglion cell loss and optic neuropathy in a humanized mouse model of familial dysautonomia. Human Molecular Genetics. 2022 Jun 1;31(11):1776-87.
[Content Brief]
Keywords
