Publication: Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β<sup>a(T87Q)</sup>-Globin Gene
Issued Date
2016-02-01
Resource Type
ISSN
15577422
10430342
10430342
Other identifier(s)
2-s2.0-84958951097
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Mahidol University
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SCOPUS
Bibliographic Citation
Human Gene Therapy. Vol.27, No.2 (2016), 148-165
Suggested Citation
Olivier Negre, Anne Virginie Eggimann, Yves Beuzard, Jean Antoine Ribeil, Philippe Bourget, Suparerk Borwornpinyo, Suradej Hongeng, Salima Hacein-Bey, Marina Cavazzana, Philippe Leboulch, Emmanuel Payen Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β<sup>a(T87Q)</sup>-Globin Gene. Human Gene Therapy. Vol.27, No.2 (2016), 148-165. doi:10.1089/hum.2016.007 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/43150
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Title
Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β<sup>a(T87Q)</sup>-Globin Gene
Abstract
© Olivier Negre et al. 2016; Published by Mary Ann Liebert, Inc. 2016. 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.