A new gene therapy for sickle celled anaemia could begin human trials next year. The therapy targets the gene BCL11A, which suppresses the production of haemoglobin. Early studies in genetically altered mice, born without the gene, pointed researchers towards this site as a potential treatment.
"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."https://www.theguardian.com/society/2016/oct/01/final-push-to-end-sickle-cell-anaemia