The Functional Effect of Repeated Cryopreservation on Transduced CD34⁺ Cells from Patients with Thalassemia.

ABSTRACT: Stable gene marking and effective engraftment of gene-modified CD34⁺ hematopoietic stem cells is a prerequisite for gene therapy success but may be challenged by the inevitable cryopreservation of the final product prior to extensive quality assurance testing. We investigated the β-globin gene transfer potency in fresh and cryopreserved CD34⁺ cells from mobilized patients with β-thalassemia, as well as the qualitative impact of repeated freeze/thaw cycles on the functionality of cultured and unmanipulated CD34⁺ cells in terms of engrafting capacity in a xenotransplantation model, under partial myeloablation. Cells transduced fresh or after one freeze-thaw cycle yielded similar clonogenic and gene transfer frequencies. Repeated cryopreservation cycles did no

SUBMITTER: Karponi G 

PROVIDER: S-EPMC6206548 | biostudies-literature | 2018 Oct

REPOSITORIES: biostudies-literature