Nature Communications (Oct 2018)

A therapeutic approach to pantothenate kinase associated neurodegeneration

  • Lalit Kumar Sharma,
  • Chitra Subramanian,
  • Mi-Kyung Yun,
  • Matthew W. Frank,
  • Stephen W. White,
  • Charles O. Rock,
  • Richard E. Lee,
  • Suzanne Jackowski

DOI
https://doi.org/10.1038/s41467-018-06703-2
Journal volume & issue
Vol. 9, no. 1
pp. 1 – 15

Abstract

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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.