Reactivation of the embryonic ζ-globin gene ameliorates severe forms of α-thalassemia.
Hemoglobinopathies are the most common inherited anemias worldwide. While most carriers are asymptomatic or mildly affected, severe forms of α-thalassemia, including severe hemoglobin H (HbH) disease and surviving individuals with Hb Bart's hydrops fetalis syndrome (BHFS), require lifelong transfusion support or stem c...
Si-Yu Liu, A. Ejaz, Andrew J. King et al.
· Nature Genetics · 0 citations