A therapeutic approach to pantothenate kinase associated neurodegeneration
Mutations in pantotenate kinase (PANK) cause neurodegneration. Here the authors carry out achemical screen and identify a PANK activator that is orally available, crosses the blood brain barrierand show that it effecttive in improving pathology and life span in a mouse model of the disease.
Main Authors: | , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Nature Publishing Group
2018-10-01
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Series: | Nature Communications |
Online Access: | https://doi.org/10.1038/s41467-018-06703-2 |