Fingolimod phosphate inhibits astrocyte inflammatory activity in mucolipidosis IV

Abstract 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...

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Veröffentlicht in:	Human molecular genetics 2018-08, Vol.27 (15), p.2725-2738
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:	Artikel
Sprache:	eng
Schlagworte:	Animals Astrocytes - drug effects Astrocytes - pathology Brain - drug effects Brain - metabolism Brain - pathology Cells, Cultured Cytokines - metabolism Disease Models, Animal Encephalitis - drug therapy Encephalitis - genetics Encephalitis - metabolism Encephalitis - pathology Female Fingolimod Hydrochloride - pharmacology Gene Expression Regulation Lysosomal Membrane Proteins - metabolism Male Mice, Knockout Mucolipidoses - drug therapy Mucolipidoses - pathology Phosphatidylinositol 3-Kinases - metabolism Phosphoproteins - metabolism Proto-Oncogene Proteins c-akt - metabolism Transient Receptor Potential Channels - genetics Transient Receptor Potential Channels - metabolism
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