Curing hemoglobinopathies: challenges and advances of conventional and new gene therapy approaches.

Inherited hemoglobin disorders, including beta-thalassemia (BT) and sickle-cell disease (SCD) are the most common monogenic diseases worldwide, with a global carrier frequency of over 5%. With migration they are becoming more common worldwide, making their management and care an increasing concern for health care systems.BT is characterized by an imbalance in the α/β-globin chain ratio, ineffective erythropoiesis, chronic hemolytic anemia, and compensatory haemopoietic expansion. Globally, there are over 25,000 births each year with transfusion-dependent thalassemia (TDT). The current available treatment for TDT is lifelong transfusions and iron chelation therapy or allogenic bone marrow as curative option. SCD affects 300 million people worldwide  and severely impacts the quality of life of patients, who experience unpredictable, recurrent acute and chronic severe pain, stroke, infections, pulmonary disease, kidney disease, retinopathy, and other complications. While survival has been dramatically extended, quality of life is markedly reduced by disease- and treatment-associated morbidity.The development of safe, tissue specific and efficient vectors, and efficient gene editing technologies have led to the development of several gene therapy trials for BT and SCD. Yet, the complexity of the approach presents its hurdles. Fundamental factors at play include the requirement for myeloablation on a patient with a benign disease, the age of the pati...
Source: Mediterranean Journal of Hematology and Infectious Diseases - Category: Hematology Source Type: research

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