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.

Bibliographic Details
Main Authors: Lalit Kumar Sharma, Chitra Subramanian, Mi-Kyung Yun, Matthew W. Frank, Stephen W. White, Charles O. Rock, Richard E. Lee, Suzanne Jackowski
Format: Article
Language:English
Published: Nature Publishing Group 2018-10-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-018-06703-2