β-globin gene transfer to human bone marrow for sickle cell disease

Autologous hematopoietic stem cell gene therapy is an approach to treating sickle cell disease (SCD) patients that may result in lower morbidity than allogeneic transplantation. We examined the potential of a lentiviral vector (LV) (CCL-βAS3-FB) encoding a human hemoglobin (HBB) gene engineered to i...

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Главные авторы: Romero, Zulema, Urbinati, Fabrizia, Geiger, Sabine, Cooper, Aaron R., Wherley, Jennifer, Kaufman, Michael L., Hollis, Roger P., Ruiz de Assin, Rafael, Senadheera, Shantha, Sahagian, Arineh, Jin, Xiangyang, Gellis, Alyse, Wang, Xiaoyan, Gjertson, David, DeOliveira, Satiro, Kempert, Pamela, Shupien, Sally, Abdel-Azim, Hisham, Walters, Mark C., Meiselman, Herbert J., Wenby, Rosalinda B., Gruber, Theresa, Marder, Victor, Coates, Thomas D., Kohn, Donald B.
Формат: Artigo
Язык:Inglês
Опубликовано: American Society for Clinical Investigation 2013
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Research Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC4011030/
https://ncbi.nlm.nih.gov/pubmed/23863630
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI67930
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