Abstract

Hemoglobinopathies 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.

Authors

Christof von Kalle, Christopher Baum, David A. Williams

×

Download this citation for these citation managers:

Or, download this citation in these formats:

If you experience problems using these citation formats, send us feedback.

Advertisement