Article
Gene Therapy of the Hemoglobinopathies
11 Sept 2020
Abstract excerpt
Abstract Sickle cell disease and the ß‐thalassemias are caused by mutations of the ß‐globin gene and represent the most frequent single gene disorders worldwide. Even in European countries with a previous low frequency of these conditions the prevalence has substantially increased following large scale migration from Africa and the Middle East to Europe. The hemoglobin diseases severely limit both, life...
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