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Christof von Kalle, Christopher Baum, David A. Williams
J Clin Invest. 2004;
114(7):889
doi:10.1172/JCI23132
Abstract |
Full text
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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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Total citations by year
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Citations to this article
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Human Gene Therapy
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Therapeutic Options for Patients with Severe ?-Thalassemia: The Need for Globin Gene Therapy
Michel Sadelain, Farid Boulad, Renzo Galanello, Patricia Giardina, Franco Locatelli, Aurelio Maggio, Stefano Rivella, Isabelle Riviere, John Tisdale
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Human Gene Therapy
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2006 |
Stem cell–derived erythroid cells mediate long-term systemic protein delivery
Alex H Chang, Matthias T Stephan, Michel Sadelain
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Nat Biotechnol
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2006 |
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