Journal
HUMAN GENE THERAPY METHODS
Volume 29, Issue 2, Pages 104-113Publisher
MARY ANN LIEBERT, INC
DOI: 10.1089/hgtb.2017.085
Keywords
CD34(+) HSC; PBSC; lentiviral vector; transduction; gene therapy
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Funding
- Heinrich Pette Institute, Leibniz Institute for Experimental Virology
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The delivery of therapeutic genes for treatment of inherited or infectious diseases frequently requires lentiviral transduction of CD34(+) hematopoietic stem and progenitor cells (HSC). Optimized transduction protocols with a therapeutic goal aim to maximize the number of transduction-positive cells while limiting the vector copy number that reach each individual cell. Importantly, the transduced HSC should maintain their stem-like properties. Here, we analyzed LentiBOOST (TM) reagent, a membrane-sealing poloxamer, with respect to enhancing lentiviral transduction of CD34(+) peripheral blood stem cells. We demonstrate that inclusion of LentiBOOST (TM) in a standard HSC transduction protocol yields high transduction efficiencies while preserving the ability of the transduced HSC to differentiate into various hematopoietic lineages. Thus, LentiBOOST (TM) reagent can significantly improve lentiviral CD34(+) HSC transduction protocols with the potential to improve production of gene-modified cell products.
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