Gene therapy approaches to hemoglobinopathies
G Ferrari, M Cavazzana… - Hematology/Oncology …, 2017 - hemonc.theclinics.com
Hemoglobinopathies are a family of inherited blood disorders characterized by the defective
synthesis of 1 of the 2 polypeptide chains of hemoglobin (a-or b-thalassemia [b-thal]) or by
the synthesis of an abnormal hemoglobin variant, such as the hemoglobin S (HbS) mutation
(bAŔE6V) that causes sickle cell diseases (SCD). Hemoglobinopathies are found all over
the world, although the carriers' relative resistance to malaria established a high frequency
of thalassemia in the Mediterranean areas, India, and the Far East, and of SCD in sub …
synthesis of 1 of the 2 polypeptide chains of hemoglobin (a-or b-thalassemia [b-thal]) or by
the synthesis of an abnormal hemoglobin variant, such as the hemoglobin S (HbS) mutation
(bAŔE6V) that causes sickle cell diseases (SCD). Hemoglobinopathies are found all over
the world, although the carriers' relative resistance to malaria established a high frequency
of thalassemia in the Mediterranean areas, India, and the Far East, and of SCD in sub …