Haematopoietic stem cell (HSC) gene therapy has demonstrated potential to treat many\ndiseases. However, current state of the art requires sophisticated ex vivo gene transfer in a\ndedicated Good Manufacturing Practices facility, limiting availability. An automated process\nwould improve the availability and standardized manufacture of HSC gene therapy. Here, we\ndevelop a novel program for semi-automated cell isolation and culture equipment to permit\ncomplete benchtop generation of gene-modified CD34�¾ blood cell products for transplantation.\nThese cell products meet current manufacturing quality standards for both mobilized\nleukapheresis and bone marrow, and reconstitute human haematopoiesis in immunocompromised\nmice. Importantly, nonhuman primate autologous gene-modified CD34�¾ cell\nproducts are capable of stable, polyclonal multilineage reconstitution with follow-up of more\nthan 1 year. These data demonstrate proof of concept for point-of-care delivery of HSC gene\ntherapy. Given the many target diseases for gene therapy, there is enormous potential for this\napproach to treat patients on a global scale.
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