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Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders

2 Oct 2019

Abstract excerpt

Abstract Hematopoietic stem cell (HSC) gene therapy is being evaluated for hemoglobin disorders including sickle cell disease (SCD). Therapeutic globin vectors have demanding requirements including high-efficiency transduction at the HSC level and high-level, erythroid-specific expression with long-term persistence. The requirement of intron 2 for high-level β-globin expression dictates a reverse-oriented...

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PMID: 31578323
DOI: 10.1038/s41467-019-12456-3

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