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Integrating HDAd5/35++ Vectors as a New Platform for HSC Gene Therapy of Hemoglobinopathies

15 Feb 2018

Abstract excerpt

We generated an integrating, CD46-targeted, helper-dependent adenovirus HDAd5/35++ vector system for hematopoietic stem cell (HSC) gene therapy. The ∼12-kb transgene cassette included a β-globin locus control region (LCR)/promoter driven human γ-globin gene and an elongation factor alpha-1 (EF1α)-mgmt P140K expression cassette, which allows for drug-controlled increase of γ-globin-expressing erythrocytes. We...

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PMID: 29766024
DOI: 10.1016/j.omtm.2018.02.004

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