Article
Phenotypic-screening generates active novel fetal globin-inducers that downregulate Bcl11a in a monkey model.
Biochemical pharmacology - 1 Jan 2020
Makino Tomohiro, Haruyama Munetada, Katayama Katsushi, Terashima Hideki, Tsunemi Tomoyuki, Miyazaki Kazuo, Terakawa Maki, Yamashiro Kyoko, Yoshioka Ryosuke, Maeda Hiroaki
Abstract excerpt
Heritable disorders associated with hemoglobin production are the most common monogenic disorders. These are mainly represented by disorders such as β-thalassemia and sickle cell disease. Induction of fetal hemoglobin (HbF) has been known to ameliorate the clinical severity of these β hemoglobinopathies. A high throughput phenotypic screening was used in this study to isolate novel compounds that may enhance the...
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