Improving Lentiviral Transduction of CD34(+) Hematopoietic Stem and Progenitor Cells
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Improving Lentiviral Transduction of CD34(+) Hematopoietic Stem and Progenitor Cells. / Hauber, Ilona; Beschorner, Niklas; Schrödel, Silke; Chemnitz, Jan; Kröger, Nicolaus; Hauber, Joachim; Thirion, Christian.
In: HUM GENE THER METHOD, Vol. 29, No. 2, 04.2018, p. 104-113.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Improving Lentiviral Transduction of CD34(+) Hematopoietic Stem and Progenitor Cells
AU - Hauber, Ilona
AU - Beschorner, Niklas
AU - Schrödel, Silke
AU - Chemnitz, Jan
AU - Kröger, Nicolaus
AU - Hauber, Joachim
AU - Thirion, Christian
PY - 2018/4
Y1 - 2018/4
N2 - 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™ reagent, a membrane-sealing poloxamer, with respect to enhancing lentiviral transduction of CD34+ peripheral blood stem cells. We demonstrate that inclusion of LentiBOOST™ 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™ reagent can significantly improve lentiviral CD34+ HSC transduction protocols with the potential to improve production of gene-modified cell products.
AB - 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™ reagent, a membrane-sealing poloxamer, with respect to enhancing lentiviral transduction of CD34+ peripheral blood stem cells. We demonstrate that inclusion of LentiBOOST™ 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™ reagent can significantly improve lentiviral CD34+ HSC transduction protocols with the potential to improve production of gene-modified cell products.
KW - Journal Article
U2 - 10.1089/hgtb.2017.085
DO - 10.1089/hgtb.2017.085
M3 - SCORING: Journal article
C2 - 29631437
VL - 29
SP - 104
EP - 113
JO - HUM GENE THER METHOD
JF - HUM GENE THER METHOD
SN - 1946-6536
IS - 2
ER -