Olivier NegreAnne Virginie EggimannYves BeuzardJean Antoine RibeilPhilippe BourgetSuparerk BorwornpinyoSuradej HongengSalima Hacein-BeyMarina CavazzanaPhilippe LeboulchEmmanuel PayenBluebird Bio, Inc.Institut des Maladies Emergentes et des Therapies InnovantesHopital Necker Enfants MaladesMahidol UniversityUniversite Paris-Sud XIBrigham and Women's HospitalInserm2018-12-112019-03-142018-12-112019-03-142016-02-01Human Gene Therapy. Vol.27, No.2 (2016), 148-16515577422104303422-s2.0-84958951097https://repository.li.mahidol.ac.th/handle/123456789/43150© 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.Mahidol UniversityBiochemistry, Genetics and Molecular BiologyReviewSCOPUS10.1089/hum.2016.007