Fingolimod phosphate inhibits astrocyte inflammatory activity in mucolipidosis IV

Mucolipidosis IV (MLIV) is an orphan neurodevelopmental disease that causes severe neurologic dysfunction and loss of vision. Currently there is no therapy for MLIV. It is caused by loss of function of the lysosomal channel mucolipin-1, also known as TRPML1. Knockout of the Mcoln1 gene in a mouse mo...

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Veröffentlicht in:Hum Mol Genet
Hauptverfasser: Weinstock, Laura D, Furness, Amanda M, Herron, Shawn S, Smith, Sierra S, Sankar, Sitara B, DeRosa, Samantha G, Gao, Dadi, Mepyans, Molly E, Scotto Rosato, Anna, Medina, Diego L, Vardi, Ayelet, Ferreira, Natalia S, Cho, Soo Min, Futerman, Anthony H, Slaugenhaupt, Susan A, Wood, Levi B, Grishchuk, Yulia
Format: Artigo
Sprache:Inglês
Veröffentlicht: Oxford University Press 2018
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Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6915831/
https://ncbi.nlm.nih.gov/pubmed/29771310
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddy182
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