Transcriptomics-informed pharmacology identifies epigenetic and cell cycle regulators that enhance AAV production.

ABSTRACT: Recombinant adeno-associated virus (rAAV) is a widely used viral vector for gene therapy. However, these vectors have limited availability due to manufacturing challenges with productivity and quality. These challenges can be addressed by better understanding the mechanisms that influence cellular responses during rAAV production. In this study, we aimed to identify targets that may enhance rAAV production using transcriptomic analyses of five cell lines with variable capacities for rAAV production. Using an intersectional approach, we measured the transcriptional responses of these cells during rAAV production and compared transcriptional profiles between high and base producers to identify possible targets for enhancing production. During rAAV production, we found transcriptional differe

SUBMITTER: Tworig J 

PROVIDER: S-EPMC11647610 | biostudies-literature | 2024 Dec

REPOSITORIES: biostudies-literature