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Proteostasis Modulation in Germline Missense von Hippel Lindau Disease

5 Apr 2023

Abstract excerpt

PURPOSE: Missense mutated von Hippel Lindau (VHL) protein (pVHL) maintains intrinsic function but undergoes proteasomal degradation and tumor initiation and/or progression in VHL disease. Vorinostat can rescue missense mutated pVHL and arrest tumor growth in preclinical models. We asked whether short-term oral vorinostat could rescue pVHL in central nervous system hemangioblastomas in patients with germline...

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PMID: 37018064
DOI: 10.1158/1078-0432.ccr-22-3651

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