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

19 Aug 2020

Abstract excerpt

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 vivo selection of transduced HSPCs. Acute innate toxicity associated with HDAd5/35++ injection was controlled by...

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PMID: 32814708
DOI: 10.1172/jci.insight.139538

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