Production and purification of high-titer foamy virus vector for the treatment of leukocyte adhesion deficiency.

ABSTRACT: Compared to other integrating viral vectors, foamy virus (FV) vectors have distinct advantages as a gene transfer tool, including their nonpathogenicity, the ability to carry larger transgene cassettes, and increased stability of virus particles due to DNA genome formation within the virions. Proof of principle of its therapeutic utility was provided with the correction of canine leukocyte adhesion deficiency using autologous CD34⁺ cells transduced with FV vector carrying the canine CD18 gene, demonstrating its long-term safety and efficacy. However, infectious titers of FV-human(h)CD18 were low and not suitable for manufacturing of clinical-grade product. Herein, we developed a scalable production and purification process that resulted in 60-fold higher FV-hCD18 titers from ~1.

SUBMITTER: Nasimuzzaman M 

PROVIDER: S-EPMC5052019 | biostudies-literature | 2016

REPOSITORIES: biostudies-literature