Streamlined rAAV HeLaS3 producer cell line generation via GS selection
The high cost and complexity of manufacturing recombinant adeno-associated virus vectors continue to limit the broader application of gene therapies, which offer life-changing potential for individuals affected by genetic diseases. Although stable producer cell lines represent a scalable and cost-effective alternative to transient transfection methods, their development is often delayed by inefficient selection strategies and extended timelines. In this study, we present a novel application of the glutamine synthetase-based selection system - commonly used in CHO cells - to a HeLaS3-based rAAV production platform. By generating glutamine synthetase-knockout HeLaS3 cells via CRISPR-Cas9 and applying glutamine deprivation under serum-free conditions, we significantly streamlined the PCL generation process, reducing the timeline to approximately two months while maintaining rAAV productivity (>1x1011 vg/mL) and product quality (~70% full capsids). This work establishes a robust and scalable workflow for rAAV manufacturing, with the potential to enhance accessibility and reduce viral vector production costs for applications in gene therapy.
Authors
- José Miguel Escandell
- Ivy Rose Sebastian (ORCID: https://orcid.org/0000-0002-9228-9029)
- Filipa Moura (ORCID: https://orcid.org/0000-0002-8765-4618)
- Mariana Antunes
- Patrícia Gomes-Alves
- Paula Alves
Institutions
- Instituto de Biologia Experimental e Tecnológica (PT)
- Universidade Nova de Lisboa (PT)
- BOKU University (AT)
Publication Details
- Journal
- Scientific Reports
- Published
- 2026-01-09
- DOI
- https://doi.org/10.1038/s41598-025-34826-2
- Primary Topic
- Viral Infectious Diseases and Gene Expression in Insects
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Austrian Science Fund
- Ministério da Educação
- Fundação para a Ciência e a Tecnologia