dc.contributor.author | Negre, Olivier | en_US |
dc.contributor.author | Eggimann, Anne-Virginie | en_US |
dc.contributor.author | Beuzard, Yves | en_US |
dc.contributor.author | Ribeil, Jean-Antoine | en_US |
dc.contributor.author | Bourget, Philippe | en_US |
dc.contributor.author | Borwornpinyo, Suparerk | en_US |
dc.contributor.author | Hongeng, Suradej | en_US |
dc.contributor.author | Hacein-Bey, Salima | en_US |
dc.contributor.author | Cavazzana, Marina | en_US |
dc.contributor.author | Leboulch, Philippe | en_US |
dc.contributor.author | Payen, Emmanuel | en_US |
dc.date.accessioned | 2016-04-01T15:48:06Z | |
dc.date.issued | 2016 | en_US |
dc.identifier.citation | Negre, 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. | en |
dc.identifier.issn | 1043-0342 | en |
dc.identifier.uri | http://nrs.harvard.edu/urn-3:HUL.InstRepos:26318640 | |
dc.description.abstract | 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. | en |
dc.language.iso | en_US | en |
dc.publisher | Mary Ann Liebert, Inc. | en |
dc.relation.isversionof | doi:10.1089/hum.2016.007 | en |
dc.relation.hasversion | http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4779296/pdf/ | en |
dash.license | LAA | en_US |
dc.title | Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-Globin Gene | en |
dc.type | Journal Article | en_US |
dc.description.version | Version of Record | en |
dc.relation.journal | Human Gene Therapy | en |
dash.depositing.author | Leboulch, Philippe | en_US |
dc.date.available | 2016-04-01T15:48:06Z | |
dc.identifier.doi | 10.1089/hum.2016.007 | * |
dash.authorsordered | false | |
dash.contributor.affiliated | Leboulch, Philippe | |