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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Autores principales: | , , , , , , , , , , , , , , |
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Formato: | article |
Lenguaje: | EN |
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Nature Portfolio
2021
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Acceso en línea: | https://doaj.org/article/f3a454c49a4a458db2c91e87d25ee520 |
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