Christof von Kalle, Christopher Baum, David A. Williams
J Clin Invest.
2004;
114(7):889–891
doi:10.1172/JCI23132
This article Copyright © 2004, The American Society for Clinical Investigation
Abstract
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H
emoglobinopathies are caused by abnormal structure or synthesis of hemoglobin chains and represent serious monogenic disorders. A new study demonstrates that lentiviral vectors can express clinically relevant levels of human transgenic β-globin in red cells of xenografted mice. While some safety concerns must be addressed, this study is an important step toward potential clinical trials of gene therapy for hemoglobinopathies.
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