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RN-1, a potent and selective lysine-specific demethylase 1 inhibitor, increases ?-globin expression, F reticulocytes, and F cells in a sickle cell disease mouse model.
The LSD1 inhibitor RN-1 recapitulates the fetal pattern of hemoglobin synthesis in baboons (P. anubis).
Oral administration of the LSD1 inhibitor ORY-3001 increases fetal hemoglobin in sickle cell mice and baboons.
Evolving treatment paradigms in sickle cell disease.
Potential role of LSD1 inhibitors in the treatment of sickle cell disease: a review of preclinical animal model data.