A synthetic platform for developing recombinant adeno-associated virus type 8 producer cell lines.

ABSTRACT: Recombinant adeno-associated virus (rAAV) is one of the most widely used viral vectors for gene therapy. It is used in very high doses for the treatment of many diseases, making large-scale production for clinical applications challenging. We have established a synthetic biology-based platform to construct stable production cell lines, which can be induced to produce rAAV2. In this study, we extended our cell line construction pipelines for rAAV2 to rAAV8, a serotype whose tropism makes it attractive for gene delivery in multiple tissues. The Genome Module, encoding the rAAV2 genome, and Replication Modules, containing Rep68, DBP and E4orf6 coding sequences, originally used for rAAV2 were retained, but the Packaging Module was modified to replace the AAV2 intron-less cap gene (VP123) with

SUBMITTER: Lin YC 

PROVIDER: S-EPMC12171320 | biostudies-literature | 2025 May-Jun

REPOSITORIES: biostudies-literature