Outcomes of Gene Therapy for Severe Sickle Cell Disease (SCD) via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral ßAT87Q-Globin Vector
Year: 2016
Type: article
Abstract: Transplantation of severe SCD patients with autologous CD34+ cells transduced with a replication-defective, self-inactivating LentiGlobin BB305 lentiviral vector containing an engineered βA-T87Q-globin gene may lead to sufficient production of anti-sickling HbA-T87Q to prevent clinical symptoms and organ damage. Herein we provide initial data on the first SCD subjects who have undergone gene therapy with the LentiGlobin BB305 vector.
Institutions: Bluebird Bio (United States), National Institutes of Health, National Institute of Diabetes and Digestive and Kidney Diseases, National Heart, Lung, and Blood Institute, Medical University of South Carolina, Université Paris-Sud, Brigham and Women's Hospital, Harvard University, Inserm, Commissariat à l'Énergie Atomique et aux Énergies Alternatives, CEA Paris-Saclay - Etablissement de Fontenay-aux-roses, Hôpital Necker-Enfants Malades
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $3,650
OpenAlex ID: W2297523524