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.

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Autores principales: Lalit Kumar Sharma, Chitra Subramanian, Mi-Kyung Yun, Matthew W. Frank, Stephen W. White, Charles O. Rock, Richard E. Lee, Suzanne Jackowski
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2018
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Acceso en línea:https://doaj.org/article/df56f7ef021a4881aab28f7777533ce7
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spelling oai:doaj.org-article:df56f7ef021a4881aab28f7777533ce72021-12-02T17:32:32ZA therapeutic approach to pantothenate kinase associated neurodegeneration10.1038/s41467-018-06703-22041-1723https://doaj.org/article/df56f7ef021a4881aab28f7777533ce72018-10-01T00:00:00Zhttps://doi.org/10.1038/s41467-018-06703-2https://doaj.org/toc/2041-1723Mutations 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.Lalit Kumar SharmaChitra SubramanianMi-Kyung YunMatthew W. FrankStephen W. WhiteCharles O. RockRichard E. LeeSuzanne JackowskiNature PortfolioarticleScienceQENNature Communications, Vol 9, Iss 1, Pp 1-15 (2018)
institution DOAJ
collection DOAJ
language EN
topic Science
Q
spellingShingle Science
Q
Lalit Kumar Sharma
Chitra Subramanian
Mi-Kyung Yun
Matthew W. Frank
Stephen W. White
Charles O. Rock
Richard E. Lee
Suzanne Jackowski
A therapeutic approach to pantothenate kinase associated neurodegeneration
description 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.
format article
author Lalit Kumar Sharma
Chitra Subramanian
Mi-Kyung Yun
Matthew W. Frank
Stephen W. White
Charles O. Rock
Richard E. Lee
Suzanne Jackowski
author_facet Lalit Kumar Sharma
Chitra Subramanian
Mi-Kyung Yun
Matthew W. Frank
Stephen W. White
Charles O. Rock
Richard E. Lee
Suzanne Jackowski
author_sort Lalit Kumar Sharma
title A therapeutic approach to pantothenate kinase associated neurodegeneration
title_short A therapeutic approach to pantothenate kinase associated neurodegeneration
title_full A therapeutic approach to pantothenate kinase associated neurodegeneration
title_fullStr A therapeutic approach to pantothenate kinase associated neurodegeneration
title_full_unstemmed A therapeutic approach to pantothenate kinase associated neurodegeneration
title_sort therapeutic approach to pantothenate kinase associated neurodegeneration
publisher Nature Portfolio
publishDate 2018
url https://doaj.org/article/df56f7ef021a4881aab28f7777533ce7
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