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Brain cell type specific proteomics approach to discover pathological mechanisms in the childhood CNS disorder mucolipidosis type IV.
Front Mol Neurosci. 2023 Aug 7;16:1215425. doi: 10.3389/fnmol.2023.1215425. eCollection 2023.
Front Mol Neurosci. 2023.
PMID: 37609073
Free PMC article.
MCOLN1 gene therapy corrects neurologic dysfunction in the mouse model of mucolipidosis IV.
DeRosa S, Salani M, Smith S, Sangster M, Miller-Browne V, Wassmer S, Xiao R, Vandenberghe L, Slaugenhaupt S, Misko A, Grishchuk Y.
DeRosa S, et al. Among authors: sangster m.
Hum Mol Genet. 2021 May 29;30(10):908-922. doi: 10.1093/hmg/ddab093.
Hum Mol Genet. 2021.
PMID: 33822942
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