1. β-globin gene transfer to human bone marrow for sickle cell disease
- Author
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Romero, Zulema, Urbinati, Fabrizia, Geiger, Sabine, Cooper, Aaron R., Wherley, Jennifer, Kaufman, Michael L., Hollis, Roger P., de Assin, Rafael Ruiz, 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., and Kohn, Donald B.
- Subjects
Care and treatment ,Research ,Genetic aspects ,Bone marrow -- Genetic aspects ,Genetic transformation -- Research ,Sickle cell anemia -- Genetic aspects -- Care and treatment ,Gene expression -- Research - Abstract
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 [...]
- Published
- 2013
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