Ultrafiltered recombinant AAV8 vector can be safely administered in vivo and efficiently transduces liver.

ABSTRACT: Viral vectors are extensively purified for use in biomedical research, in order to separate biologically active virus particles and to eliminate production related impurities that are assumed to be detrimental to the host. For recombinant adeno-associated virus (rAAV) vectors this is typically accomplished using density gradient-based methods, which are tedious and require specialized ultracentrifugation equipment. In order to streamline the preparation of rAAV vectors for pilot and small animal studies, we recently devised a simple ultrafiltration approach that permits rapid virus concentration and partial removal of production-related impurities. Here we show that systemic administration of such rapidly prepared (RP) rAAV8 vectors in mice is safe and efficiently transduces the liver. Acr

SUBMITTER: Kleven MD 

PROVIDER: S-EPMC5886455 | biostudies-literature | 2018

REPOSITORIES: biostudies-literature