DNA Minicircle Technology Improves Purity of Adeno-associated Viral Vector Preparations.

ABSTRACT: Adeno-associated viral (AAV) vectors are considered as one of the most promising delivery systems in human gene therapy. In addition, AAV vectors are frequently applied tools in preclinical and basic research. Despite this success, manufacturing pure AAV vector preparations remains a difficult task. While empty capsids can be removed from vector preparations owing to their lower density, state-of-the-art purification strategies as of yet failed to remove antibiotic resistance genes or other plasmid backbone sequences. Here, we report the development of minicircle (MC) constructs to replace AAV vector and helper plasmids for production of both, single-stranded (ss) and self-complementary (sc) AAV vectors. As bacterial backbone sequences are removed during MC production, encapsidation of pro

SUBMITTER: Schnodt M 

PROVIDER: S-EPMC5023404 | biostudies-literature | 2016

REPOSITORIES: biostudies-literature