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BPN15477

Chemical Structure : BPN15477

CAS No.: 1971086-99-3

BPN15477 (BPN-15477)

货号: PC-49818Not For Human Use, Lab Use Only.

BPN-15477 (BPN15477) is a potent splicing modulator compound that restores correct splicing of ELP1 exon 20, increases full-length ELP1 mRNA by increasing exon 20 inclusion with EC50 of 1.9 uM in luciferase splicing assays.

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纯度 & COA & 质检文件 纯度: >98% (HPLC) Select Batch:

生物&药学活性

BPN-15477 (BPN15477) is a potent splicing modulator compound that restores correct splicing of ELP1 exon 20, increases full-length ELP1 mRNA by increasing exon 20 inclusion with EC50 of 1.9 uM in luciferase splicing assays.
BPN-15477 is significantly more potent and efficacious than kinetin, and BPN-15477 modulates splicing selectively.
BPN-15477 corrects splicing of the ELP1 transcript, significantly increases the level of functional protein in vivo in all tissues, including brain.
BPN-15477 treatment increases LIPA and CFTR protein in disease-relevant cellular models.
BPN-15477 treatment (70 mg/Kg/day) improves exon 20 inclusion in the retina and brain of mice carrying the human FD transgene TgFD9.

物理化学性质&存储条件

分子量 259.70
分子式 C12H10ClN5
外观性状 Solid
CAS No.
储存条件
固体粉末
-20°C 12 个月; 4°C 6 个月
配置液
-80°C 6 个月; -20°C 6 个月
Shipping
Solubility

10 mM in DMSO

Chemical Name/SMILES

2-Chloro-N-(4-pyridinylmethyl)-7H-pyrrolo[2,3-d]pyrimidin-4-amine

参考文献

1. Dadi Gao, et al. Nat Commun. 2021 Jun 7;12(1):3332.

2. Salani, M. et al. SLAS Discov.24, 57–67 (2018).

3. Morini E, et al. J Genet Genomics. 2022 Jul;49(7):654-665.

4. Anil Chekuri, et al. Hum Mol Genet. 2022 Jun 4;31(11):1776-1787.

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