In vivo HSC transduction in humanized mice mediated by novel capsid-modified HDAd vectors

We developed an in vivo hematopoietic stem cell (HSC) gene therapy approach consisting of HSC mobilization and intravenous injection of helper dependent adenovirus (HDAd) vectors. While we have demonstrated safety and efficacy of the in vivo approach in CD46-transgenic mice and rhesus macaques, stud...

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Bibliographic Details
Main Authors: Aphrodite Georgakopoulou, Hongjie Wang, Jiho Kim, Chang Li, André Lieber
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Methods & Clinical Development
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Online Access:http://www.sciencedirect.com/science/article/pii/S2329050125000439
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