Oral Administration of the LSD1 Inhibitor ORY-3001 Increases Fetal Hemoglobin in Sickle Cell Mice and Baboons

The β-hemoglobinopathies, consisting of sickle cell disease (SCD) and the β-thalassemias, are the most common genetic diseases worldwide. These syndromes cause widespread suffering and significant reduction in lifespan in patients. The β-thalassemia syndromes comprise a spectrum of disorders characte rized by deficiencies in the production of the adult β-globin chain [1]. Increased expression of the fetal γ-globin chains and Fetal Hemoglobin [(HbF(α2γ2)] can augment reduced β-globin chain deficiencies and is therefore considered a desirable therapeutic target that can ameliorate the symptom s of this disease [2,3].
Source: Experimental Hematology - Category: Hematology Authors: Tags: Brief Communication Source Type: research