Accurate Quantification and Characterization of Adeno-Associated Viral Vectors.

ABSTRACT: One of the main challenges in the gene therapy viral vector development is to establish an optimized process for its large scale production. This requires optimization for upstream and downstream processes as well as methods that enable the step-by step analytical characterization of the virus, the results of which inform the iterative refinement of production for yield, purity and potency. The biggest problem here is a plethora of viral vector formulations, many of which interfere with analytical techniques. We took adeno-associated virus (AAV) as an example and showed benefits of combined use of molecular methods and transmission electron microscopy (TEM) for viral vectors' characterization and quantification. Results of the analyses showed that droplet digital PCR (ddPCR) performs bette

SUBMITTER: Dobnik D 

PROVIDER: S-EPMC6650692 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature