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Efficient correction of hemoglobinopathy-causing mutations by homologous recombination in integration-free patient iPSCs
Mo Li, Keiichiro Suzuki, Jing Qu, Preeti Saini, Ilir Dubova, Fei Yi, Jungmin Lee, Ignacio Sancho-Martinez, Guang-Hui Liu and Juan Carlos Izpisua Belmonte
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.