Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors

Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors

We tested a new in vivo hematopoietic stem cell (HSC) transduction/selection approach in rhesus macaques using HSC-tropic, integrating, helper-dependent adenovirus vectors (HDAd5/35++) designed for the expression of human γ-globin in red blood cells (RBCs) to treat hemoglobinopathies. We show that H...

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Bibliographic Details
Main Authors: Chang Li, Hongjie Wang, Sucheol Gil, Audrey Germond, Connie Fountain, Audrey Baldessari, Jiho Kim, Zhinan Liu, Aphrodite Georgakopoulou, Stefan Radtke, Tamás Raskó, Amit Pande, Christina Chiang, Eli Chin, Evangelia Yannaki, Zsuzsanna Izsvák, Thalia Papayannopoulou, Hans-Peter Kiem, André Lieber
Format: Article
Language:English
Published: Elsevier 2022-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
in vivo
hematopoietic stem cells
adenovirus vector
nonhuman primates
hemoglobinopathies
gamma globin
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121001893

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http://www.sciencedirect.com/science/article/pii/S2329050121001893

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