Blood phenylalanine reduction reverses gene expression changes observed in a mouse model of phenylketonuria

Blood phenylalanine reduction reverses gene expression changes observed in a mouse model of phenylketonuria

Abstract Phenylketonuria (PKU) is a genetic deficiency of phenylalanine hydroxylase (PAH) in liver resulting in blood phenylalanine (Phe) elevation and neurotoxicity. A pegylated phenylalanine ammonia lyase (PEG-PAL) metabolizing Phe into cinnamic acid was recently approved as treatment for PKU pati...

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Main Authors: Rachna Manek, Yao V. Zhang, Patricia Berthelette, Mahmud Hossain, Cathleen S. Cornell, Joseph Gans, Gulbenk Anarat-Cappillino, Sarah Geller, Robert Jackson, Dan Yu, Kuldeep Singh, Sue Ryan, Dinesh S. Bangari, Ethan Y. Xu, Sirkka R. M. Kyostio-Moore
Format: article
Language:EN
Published: Nature Portfolio 2021
Subjects:
Medicine
R
Science
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Online Access:https://doaj.org/article/f3a454c49a4a458db2c91e87d25ee520
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