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<i>MLC1</i> alteration in iPSCs give rise to disease-like cellular vacuolation phenotype in the astrocyte lineage

2025-01-08

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Background</h4> Megalencephalic leukoencephalopathy with subcortical cysts (MLC), a rare and progressive neurodegenerative disorder involving the white matter, is not adequately recapitulated by current disease models. Somatic cell reprogramming, along with advancements in genome engineering, may allow the establishment of in-vitro human models of MLC for disease modeling and drug screenin...

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Literature Corpus work
44cfff68-ff06-5385-9a7f-e88ed95cd207
DOI
10.1101/2025.01.06.631607
Open publication

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<i>MLC1</i> alteration in iPSCs give rise to disease-like cellular vacuolation phenotype in the astrocyte lineageDOI 10.1101/2025.01.06.631607
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