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

Authors :: 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.
Kohn, Donald B.
Source :: Journal of Clinical Investigation. August 1, 2013, Vol. 123 Issue 8, p3317, 14 p.
Publication Year :: 2013
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 [...]

Subjects :: Sickle cell anemia -- Genetic aspects -- Care and treatment
Gene expression -- Research
Bone marrow -- Genetic aspects
Genetic transformation -- Research
Health care industry

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