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Usage Information

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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Commentary Article has an altmetric score of 3

Lenti in red: progress in gene therapy for human hemoglobinopathies

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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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Usage data is cumulative from May 2024 through May 2025.

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Figure 48 1
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