October 3, 2016
(The Guardian) – Scientists are finalising plans to use gene therapy to treat one of the world’s most widespread inherited diseases – sickle cell anaemia. The technique could begin trials next year, say researchers. About 300,000 babies are born globally with sickle cell disease. The condition causes red blood cells to deform, triggering anaemia, pain, organ failure, tissue damage, strokes and heart attacks. In the west, patients now live to their 40s thanks to the availability of blood transfusions and other treatments. But in Africa most still die in childhood.