Assessment of the percentage of full recombinant adeno-associated virus particles in a gene therapy drug using CryoTEM.

ABSTRACT: In spite of continuous development of gene therapy vectors with thousands of drug candidates in clinical drug trials there are only a small number approved on the market today stressing the need to have characterization methods to assist in the validation of the drug development process. The level of packaging of the vector capsids appears to play a critical role in immunogenicity, hence an objective quantitative method assessing the content of particles containing a genome is an essential quality measurement. As transmission electron microscopy (TEM) allows direct visualization of the particles present in a specimen, it naturally seems as the most intuitive method of choice for characterizing recombinant adeno-associated virus (rAAV) particle packaging. Negative stain TEM (nsTEM) is an es

SUBMITTER: Colomb-Delsuc M 

PROVIDER: S-EPMC9165851 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature