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Cell Res. 2013 Aug;23(8):975-7. doi: 10.1038/cr.2013.73. Epub 2013 Jun 4.

Differentiation therapy for IDH1/2 mutant malignancies.

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Department of Medicine, Division of Hematology/Oncology, Weill Cornell Medical College, 1300 York Ave, New York, NY 10065, USA.


Recently discovered recurrent somatic mutations in the key metabolic enzymes IDH1 and IDH2 produce the aberrant oncometabolite 2-HG and contribute to malignant transformation of hematopoietic and glial cells. Two recent reports in Science describe the first IDH1 and IDH2 mutant-specific small-molecule inhibitors, which induce cell differentiation of myeloid leukemias and malignant gliomas.

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