Efficient correction of hemoglobinopathy-causing mutations by homologous recombination in integration-free patient iPSCs

摘要：

Dear Editor,Hemoglobinopathies are a collection of heritable diseases caused by abnormal structure or insufficient production of hemoglobins ”1”.Many forms of hemoglobinopathies,such as sickle cell disease (SCD) and β-thalassemia,which are caused by mutations of the β-globin (HBB) gene,lead to severe anemia and other life-threatening conditions.Ultimately,due to the lack of a curative treatment,patients generally have shortened life spans even under life-long supportive care.Given the high prevalence of these diseases,especially in some areas of Africa,Asia and the Mediterranean region,much effort has been devoted to developing a genetic cure for hemoglobinopathies.

机标关键词：curative treatment、especially、collection、structure、region、effort、lack、high、cell、SCD

所属期刊栏目：21

分类号：R329.2(人体形态学)

在线出版日期：2012-03-20（万方平台首次上网日期，不代表论文的发表时间）

页码：1740-1744