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BPN-15477 

Cat. No. M29069

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BPN-15477  Structure

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Quality Control & Documentation
Biological Activity

BPN-15477 is a potent SMC (splicing modulator compound) that restores correct splicing of ELP1 (Elongator complex protein 1) exon 20. BPN-15477 corrects splicing of the ELP1 transcript, significantly increases the level of functional protein in vivo in all tissues, including brain. BPN-15477 can be used for frontotemporal dementia research.

Chemical Information
Molecular Weight 259.69
Formula C12H10ClN5
CAS Number 1971086-99-3
Form Solid
Solubility (25°C) DMSO 25 mg/mL (ultrasonic)
Storage Powder          -20°C   3 years ;  4°C   2 years
In solvent       -80°C   6 months ;  -20°C   1 month
References

[1] Elisabetta Morini, et al. Am J Hum Genet. Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia

[2] Giulia Romano, et al. Am J Hum Genet. Rescue of a familial dysautonomia mouse model by AAV9-Exon-specific U1 snRNA

[3] Anil Chekuri, et al. Hum Mol Genet. Selective retinal ganglion cell loss and optic neuropathy in a humanized mouse model of familial dysautonomia

[4] Elisabetta Morini, et al. J Genet Genomics. Developmental regulation of neuronal gene expression by Elongator complex protein 1 dosage

[5] Dadi Gao, et al. Nat Commun. A deep learning approach to identify gene targets of a therapeutic for human splicing disorders

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Keywords: BPN-15477  supplier, inhibitors, activators

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