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Title: Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β<sup>a(T87Q)</sup>-Globin Gene
Authors: 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
Bluebird Bio, Inc.
Institut des Maladies Emergentes et des Therapies Innovantes
Hopital Necker Enfants Malades
Mahidol University
Universite Paris-Sud XI
Brigham and Women's Hospital
Keywords: Biochemistry, Genetics and Molecular Biology
Issue Date: 1-Feb-2016
Citation: Human Gene Therapy. Vol.27, No.2 (2016), 148-165
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.
ISSN: 15577422
Appears in Collections:Scopus 2016-2017

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