Gene Therapy Can Help Over 330,000 Children Each Year Born With Hemoglobinopathies

Cleveland Clinic

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Hemoglobinopathies are genetic disorders affecting the structure or production of the hemoglobin molecule – the red protein responsible for transporting oxygen in the blood.

The most common hemoglobinopathies include sickle cell disease and thalassemia – which combined affect more than 330,000 children born worldwide every year and more than 100,000 patients with sickle cell disease in the United States alone.

The latest research in hemoglobinopathies has brought an experimental gene therapy, giving those who have the condition the potential ability to make functional hemoglobin molecules – reducing the presence of sickled blood cells or ineffective red blood cells in thalassemia to prevent associated complications.