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Nat Med. 2013 Mar;19(3):291-4. doi: 10.1038/nm.3101. Epub 2013 Feb 17.

Lysine-specific demethylase 1 is a therapeutic target for fetal hemoglobin induction.

Author information

1
Department of Cell and Developmental Biology, University of Michigan Medical School, Ann Arbor, Michigan, USA.

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.

PMID:
23416702
PMCID:
PMC5512162
DOI:
10.1038/nm.3101
[Indexed for MEDLINE]
Free PMC Article

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