AAV-p40 Bioengineering Platform for Variant Selection Based on Transgene Expression.

ABSTRACT: The power of adeno-associated viral (AAV)-directed evolution for identifying novel vector variants with improved properties is well established, as evidenced by numerous publications reporting novel AAV variants. However, most capsid variants reported to date have been identified using either replication-competent (RC) selection platforms or polymerase chain reaction-based capsid DNA recovery methods, which can bias the selection toward efficient replication or unproductive intracellular trafficking, respectively. A central objective of this study was to validate a functional transduction (FT)-based method for rapid identification of novel AAV variants based on AAV capsid mRNA expression in target cells. We performed a comparison of the FT platform with existing RC strategies. Based on the

SUBMITTER: Westhaus A 

PROVIDER: S-EPMC10112876 | biostudies-literature | 2022 Jun

REPOSITORIES: biostudies-literature