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|Title:||Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β<sup>a(T87Q)</sup>-Globin Gene|
Anne Virginie Eggimann
Jean Antoine Ribeil
Bluebird Bio, Inc.
Institut des Maladies Emergentes et des Therapies Innovantes
Hopital Necker Enfants Malades
Universite Paris-Sud XI
Brigham and Women's Hospital
|Keywords:||Biochemistry, Genetics and Molecular Biology|
|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.|
|Appears in Collections:||Scopus 2016-2017|
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