Modulation of Human Phenylalanine Hydroxylase by 3-Hydroxyquinolin-2(1H)-One Derivatives

نویسندگان

چکیده

Phenylketonuria (PKU) is a genetic disease caused by deficient activity of human phenylalanine hydroxylase (hPAH) that, when untreated, can lead to severe psychomotor impairment. Protein misfolding recognized as the main underlying pathogenic mechanism PKU. Therefore, use stabilizers protein structure and/or an attractive therapeutic strategy for this condition. Here, we report that 3-hydroxyquinolin-2(1H)-one derivatives act protectors hPAH enzyme activity. Electron paramagnetic resonance spectroscopy demonstrated compounds affect coordination non-heme ferric center at active-site. Moreover, surface plasmon studies showed these stabilizing be outcompeted natural substrate l-phenylalanine. Two designed functionally stabilized maintaining This effect was observed on recombinant purified and in cellular model. Besides interacting with catalytic iron, one also binds N-terminal regulatory domain, although different location from allosteric l-Phe binding site, supported solution structures obtained small-angle X-ray scattering.

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ژورنال

عنوان ژورنال: Biomolecules

سال: 2021

ISSN: ['2218-273X']

DOI: https://doi.org/10.3390/biom11030462