Practical utilization of recombinant AAV vector reference standards: focus on vector genomes titration by free ITR qPCR.

ABSTRACT: Clinical trials using recombinant adeno-associated virus (rAAV) vectors have demonstrated efficacy and a good safety profile. Although the field is advancing quickly, vector analytics and harmonization of dosage units are still a limitation for commercialization. AAV reference standard materials (RSMs) can help ensure product safety by controlling the consistency of assays used to characterize rAAV stocks. The most widely utilized unit of vector dosing is based on the encapsidated vector genome. Quantitative polymerase chain reaction (qPCR) is now the most common method to titer vector genomes (vg); however, significant inter- and intralaboratory variations have been documented using this technique. Here, RSMs and rAAV stocks were titered on the basis of an inverted terminal repeats (ITRs)

SUBMITTER: D'Costa S 

PROVIDER: S-EPMC4813604 | biostudies-literature | 2016

REPOSITORIES: biostudies-literature