Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy.

ABSTRACT: Haematopoietic stem cell (HSC) gene therapy has demonstrated potential to treat many diseases. However, current state of the art requires sophisticated ex vivo gene transfer in a dedicated Good Manufacturing Practices facility, limiting availability. An automated process would improve the availability and standardized manufacture of HSC gene therapy. Here, we develop a novel program for semi-automated cell isolation and culture equipment to permit complete benchtop generation of gene-modified CD34⁺ blood cell products for transplantation. These cell products meet current manufacturing quality standards for both mobilized leukapheresis and bone marrow, and reconstitute human haematopoiesis in immunocompromised mice. Importantly, nonhuman primate autologous gene-modified CD34

SUBMITTER: Adair JE 

PROVIDER: S-EPMC5080442 | biostudies-literature | 2016 Oct

REPOSITORIES: biostudies-literature