Delivery of large transgene cassettes by foamy virus vector.

ABSTRACT: Viral vectors are effective tools in gene therapy, but their limited packaging capacity can be restrictive. Larger clinically-relevant vectors are needed. Foamy viruses have the largest genomes among mammalian retroviruses and their vectors have shown potential for gene therapy in preclinical studies. However, the effect of vector genome size on titre has not been determined. We inserted increasing lengths of the dystrophin open reading frame in a foamy virus vector and quantified packaged vector RNA and integrated DNA. For both measures, a semi-logarithmic reduction in titre was observed as genome size increased. Concentrated titres were reduced 100-fold to approximately 10⁶ transducing units per ml when vector genomes harboured a 12 kb insert, approximately twice that reported

SUBMITTER: Sweeney NP 

PROVIDER: S-EPMC5556010 | biostudies-literature | 2017 Aug

REPOSITORIES: biostudies-literature