Erythroid differentiation and viability remain unaffected in human CD34⁺ cells treated with HDAC1 degrader ARP-47, suggesting a new approach to β-hemoglobinopathies.
Key Points
ARP-47 treatment increased HbF production significantly while maintaining cell viability and differentiation.
At 10 nM, HbF levels rose significantly compared to control—with a 2.5-fold increase in HBG1/2 mRNA.
Assessment was done via flow cytometry, qPCR, and Western blot in erythroid differentiation experiments.
Selective HDAC1 degradation may lead to safer therapies for conditions like sickle cell disease.