Article

Identification of repurposable cytoprotective drugs for Vanishing White Matter Disease

2020-06-03

Abstract excerpt

Vanishing white matter disease (VWMD) is a rare leukodystrophy involving loss of function mutations of the guanine exchange factor eIF2B and typically presenting with juvenile onset. We aimed to identify repurposable FDA approved drugs in an in vitro drug screen using patient-derived fibroblasts and induced pluripotent stem cell (iPSC)-derived astrocytes. Dysregulated GADD34 and CHOP were identified in patient fi...

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Literature Corpus work: adc10874-8eed-50e0-bfab-0b37fb1f404c
DOI: 10.1101/2020.06.02.131052

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