Characterization of AAV vectors: A review of analytical techniques and critical quality attributes.

ABSTRACT: Standardized evaluation of adeno-associated virus (AAV) vector products for biotherapeutic application is essential to ensure the safety and efficacy of gene therapies. This includes analyzing the critical quality attributes of the product. However, many of the current analytical techniques used to assess these attributes have limitations, including low throughput, large sample requirements, poorly understood measurement variability, and lack of comparability between methods. To address these challenges, it is essential to establish higher-order reference methods that can be used for comparability measurements, optimization of current assays, and development of reference materials. Highly precise methods are necessary for measuring the empty/partial/full capsid ratios and the titer of AAV

SUBMITTER: Kontogiannis T 

PROVIDER: S-EPMC11372808 | biostudies-literature | 2024 Sep

REPOSITORIES: biostudies-literature