Hemoglobin disorders: lentiviral gene therapy in the starting blocks to enter clinical practice

The β-hemoglobinopathies, β-thalassemia (β-thal) and sickle cell disease (SCD), are the most common monogenic diseases worldwide. They result from mutations in the β-globin (HBB) gene locus that lead to the production of insufficient (β0, β+) or aberrant (βS) β-globin protein.

Source: Experimental Hematology - May 26, 2018 Category: Hematology Authors: Karine Sii-Felice, Marie Giorgi, Philippe Leboulch, Emmanuel Payen Source Type: research