Cell-based Genetic Therapy for the Induction of Foetal Haemoglobin in Sickle Cell Disease and Transfusion-dependent β-thalassaemia
Martin H Steinberg
Mutations of the β-globin gene (HBB) cause sickle cell disease and β-thalassaemia, collectively named the β-haemoglobinopathies. As mankind’s most common severe genetic diseases, where they are most prevalent, public health systems can be stressed.1–3 Asymptomatic heterozygous carriers are protected from severe P. falciparum infection. This has allowed the causative genes to reach polymorphic frequencies in some tropical […]