Lysine-specific demethylase 1 is a therapeutic target for fetal hemoglobin induction.
basic_science · Level V
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- Record sourced from PubMed, PMID 23416702.
- Also identified by DOI 10.1038/nm.3101 and PMC identifier 5512162.
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Abstract
Enhanced fetal γ-globin synthesis alleviates symptoms of β-globinopathies such as sickle cell disease and β-thalassemia, but current γ-globin-inducing drugs offer limited beneficial effects. We show here that lysine-specific demethylase 1 (LSD1) inhibition by RNAi in human erythroid cells or by the monoamine oxidase inhibitor tranylcypromine in human erythroid cells or β-type globin-transgenic mice enhances γ-globin expression. LSD1 is thus a promising therapeutic target for γ-globin induction, and tranylcypromine may serve as a lead compound for the development of a new γ-globin inducer.
Medical subject headings
- Fetal Hemoglobin
- Histone Demethylases
- Monoamine Oxidase Inhibitors
- Tranylcypromine