Citations to this article

Lenti in red: progress in gene therapy for human hemoglobinopathies
Christof von Kalle, … , Christopher Baum, David A. Williams
Christof von Kalle, … , Christopher Baum, David A. Williams
Published October 1, 2004
Citation Information: J Clin Invest. 2004;114(7):889-891. https://doi.org/10.1172/JCI23132.
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Lenti in red: progress in gene therapy for human hemoglobinopathies

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

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Year: 2009 2008 Total
Citations: 5 1 6
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Citations to this article (6)

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A Perumbeti, T Higashimoto, F Urbinati, R Franco, HJ Meiselman, D Witte, P Malik 		Blood 2009
Genotoxic Potential of Lineage-specific Lentivirus Vectors Carrying the β-Globin Locus Control Region
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