Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements

Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements

Recently, we demonstrated that hematopoietic stem/progenitor cell (HSPC) mobilization followed by intravenous injection of integrating, helper-dependent adenovirus HDAd5/35++ vectors resulted in efficient transduction of long-term repopulating cells and disease amelioration in mouse models after in...

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Bibliographic Details
Main Authors: Hongjie Wang, Aphrodite Georgakopoulou, Chang Li, Zhinan Liu, Sucheol Gil, Ashvin Bashyam, Evangelia Yannaki, Achilles Anagnostopoulos, Amit Pande, Zsuzsanna Izsvák, Thalia Papayannopoulou, André Lieber
Format: Article
Language:English
Published: American Society for Clinical investigation 2020-08-01
Series:JCI Insight
Subjects:
Hematology
Therapeutics
Online Access:https://doi.org/10.1172/jci.insight.139538

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