Published in
American Society of Hematology, Blood Advances, 19(3), p. 2883-2894, 2019
DOI: 10.1182/bloodadvances.2019000706
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High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells
Journal article published in 2019 by
Hongjie Wang,
Zhinan Liu,
Chang Li,
Sucheol Gil,
Thalia Papayannopoulou,
Christopher B. Doering,
André Lieber
This paper is made freely available by the publisher.
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Abstract
Key Points An in vivo HSC transduction/selection allows for high-level protein expression from erythroid cells without side effects on erythropoiesis. This approach that did not require ex vivo HSC manipulation and transplantation resulted in phenotypic correction of murine hemophilia A.