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Your search keyword '"Leboulch, Philippe"' showing total 4 results
Start Over Author "Leboulch, Philippe" Publisher mary ann liebert, inc.
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3. Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-Globin Gene

Author

Negre, Olivier, Eggimann, Anne-Virginie, Beuzard, Yves, Ribeil, Jean-Antoine, Bourget, Philippe, Borwornpinyo, Suparerk, Hongeng, Suradej, Hacein-Bey, Salima, Cavazzana, Marina, Leboulch, Philippe, and Payen, Emmanuel

Abstract

β-globin gene disorders are the most prevalent inherited diseases worldwide and result from abnormal β-globin synthesis or structure. Novel therapeutic approaches are being developed in an effort to move beyond palliative management. Gene therapy, by ex vivo lentiviral transfer of a therapeutic β-globin gene derivative (βAT87Q-globin) to hematopoietic stem cells, driven by cis-regulatory elements that confer high, erythroid-specific expression, has been evaluated in human clinical trials over the past 8 years. βAT87Q-globin is used both as a strong inhibitor of HbS polymerization and as a biomarker. While long-term studies are underway in multiple centers in Europe and in the United States, proof-of-principle of efficacy and safety has already been obtained in multiple patients with β-thalassemia and sickle cell disease.

Published
2016
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