Quantitative analysis of genome packaging in recombinant AAV vectors by charge detection mass spectrometry.

ABSTRACT: Recombinant adeno-associated virus (rAAV) has emerged as an important gene therapy vector with many clinical trials currently in progress. Analytical characterization and quantitation of particle content remain challenges in both the development and production of rAAV vectors. In this study, charge detection mass spectrometry (CDMS) and gel electrophoresis are used to characterize the DNA content of recombinant AAV8 (rAAV8) vectors with a wide range of target genome sizes. We show that the differences between the masses of empty particles and particles with the genome of interest (GOI) are correlated with the expected genome mass. A small systematic deviation (around 2%) is attributed to the packaging of counterions along with the DNA. In addition to the GOI, a broad distribution of hetero

SUBMITTER: Barnes LF 

PROVIDER: S-EPMC8476707 | biostudies-literature | 2021 Dec

REPOSITORIES: biostudies-literature