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DHODH inhibition synergizes with DNA-demethylating agents in the treatment of myelodysplastic syndromes

20 Jan 2021

Abstract excerpt

Dihydroorotate dehydrogenase (DHODH) catalyzes a rate-limiting step in de novo pyrimidine nucleotide synthesis. DHODH inhibition has recently been recognized as a potential new approach for treating acute myeloid leukemia (AML) by inducing differentiation. We investigated the efficacy of PTC299, a novel DHODH inhibitor, for myelodysplastic syndrome (MDS). PTC299 inhibited the proliferation of MDS cell lines, and...

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PMID: 33496740
DOI: 10.1182/bloodadvances.2020001461

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