Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-Globin Gene
Payen, EmmanuelNote: Order does not necessarily reflect citation order of authors.
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CitationNegre, O., A. Eggimann, Y. Beuzard, J. Ribeil, P. Bourget, S. Borwornpinyo, S. Hongeng, et al. 2016. “Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-Globin Gene.” Human Gene Therapy 27 (2): 148-165. doi:10.1089/hum.2016.007. http://dx.doi.org/10.1089/hum.2016.007.
Abstractβ-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.
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