Engineering Organoids for in vitro Modeling of Phenylketonuria

Engineering Organoids for in vitro Modeling of Phenylketonuria

Phenylketonuria is a recessive genetic disorder of amino-acid metabolism, where impaired phenylalanine hydroxylase function leads to the accumulation of neurotoxic phenylalanine levels in the brain. Severe cognitive and neuronal impairment are observed in untreated/late-diagnosed patients, and even...

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Bibliographic Details
Main Authors: Alice C. Borges, Kerensa Broersen, Paula Leandro, Tiago G. Fernandes
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-01-01
Series:Frontiers in Molecular Neuroscience
Subjects:
phenylketonuria
human induced pluripotent stem cells
organoids
neurodegeneration
neurodevelopment
disease modeling
Online Access:https://www.frontiersin.org/articles/10.3389/fnmol.2021.787242/full

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https://www.frontiersin.org/articles/10.3389/fnmol.2021.787242/full

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