| Literature DB >> 23416702 |
Lihong Shi1, Shuaiying Cui, James D Engel, Osamu Tanabe.
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.Entities:
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Year: 2013 PMID： 23416702 PMCID： PMC5512162 DOI： 10.1038/nm.3101
Source DB: PubMed Journal: Nat Med ISSN： 1078-8956 Impact factor: 53.440