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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Detalhes bibliográficos
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2013
Assuntos:
Research Article
Acesso em linha: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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