Lentiviral Gene Transfer Improves Human Alpha Globin Production
Researchers at Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine pioneered a new model that offers a potential platform for developing novel therapies to treat Alpha Thalassemia (AT), a severe blood disorder. The findings were published in the journal Blood. Thousands of children are born with AT every year, especially in South-East Asia, India, the Middle East, and the Mediterranean basin. When functioning normally, the genetic trait may provide protection against malaria and result in mild anemia. However, when both parents are carriers of faulty genes, children face an elevated risk of severe AT. In the most severe cases, the disease can be fatal without in utero intervention. Children with severe AT often require ongoing blood transfusions and extensive medical care. Allogeneic bone marrow transplantation (BMT) is currently the only available therapeutic option for…



