Article

Patient-Derived Cellular Models for Polytarget Precision Medicine in Pantothenate Kinase-Associated Neurodegeneration

2023-09-22

Abstract excerpt

The term neurodegeneration with brain iron accumulation (NBIA) brings together a broad set of progressive and disabling neurological genetic disorders in which iron is deposited preferentially in certain areas of the brain. Among NBIA disorders, the most frequent subtype is pantothenate kinase-associated neurodegeneration (PKAN) caused by pathologic variants in the PANK2 gene codifying the enzyme pantothenate kina...

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Literature Corpus work: 8e4df53d-0bf5-58fc-8e9e-2b8912d7148c
DOI: 10.20944/preprints202309.1526.v1

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