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An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

3 Aug 2018

Abstract excerpt

Autologous transplantation of hematopoietic stem cells transduced with a lentiviral vector (LV) expressing an anti-sickling HBB variant is a potential treatment for sickle cell disease (SCD). With a clinical trial as our ultimate goal, we generated LV constructs containing an anti-sickling HBB transgene ( HBBAS3 ), a minimal HBB promoter, and different combinations of DNase I hypersensitive sites (HSs) from the...

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PMID: 30140714
DOI: 10.1016/j.omtm.2018.07.012

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