2020
DOI: 10.21203/rs.3.rs-48008/v1
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Identification of Repurposable Cytoprotective Drugs in Vanishing White Matter Disease Patient-Derived cells

Abstract: BackgroundVanishing white matter disease (VWMD) is a rare leukodystrophy caused by mutations of the guanine exchange factor eIF2B that typically presents with juvenile onset. There are few treatments and no cures for the disease. Recent progress in the field has established mitochondrial dysfunction and endoplasmic reticulum (ER) stress to be strongly implicated in observed glial cell pathology. Drug repurposing offers a rapid approach toward translation of therapeutics with already-licensed drugs. ObjectiveTh… Show more

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“…A panel of candidate drugs, including berberine, defl azacort, ursodiol, zileuton, guanabenz and Anavex 2-73, and preclinical ISRIB (integrated stress response inhibitor), increased cell survival of EIF2B5 R113H/A403V or EIF2B2 G200V/E213G VWMD astrocytes, and were further investigated for their effect on the integrated stress response and mitochondrial stress [30].…”
Section: Commentmentioning
confidence: 99%
“…A panel of candidate drugs, including berberine, defl azacort, ursodiol, zileuton, guanabenz and Anavex 2-73, and preclinical ISRIB (integrated stress response inhibitor), increased cell survival of EIF2B5 R113H/A403V or EIF2B2 G200V/E213G VWMD astrocytes, and were further investigated for their effect on the integrated stress response and mitochondrial stress [30].…”
Section: Commentmentioning
confidence: 99%