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.

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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

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article

Streamlined rAAV HeLaS3 producer cell line generation via GS selection

José Miguel Escandell, Ivy Rose Sebastian, Filipa Moura, Mariana Antunes et al.
Scientific Reports
Viral Infectious Diseases and Gene Expression in Insects
article

Streamlined rAAV HeLaS3 producer cell line generation via GS selection

José Miguel Escandell, Ivy Rose Sebastian, Filipa Moura, Mariana Antunes, Patrícia Gomes-Alves, Paula Alves
article en

Abstract

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.

Scientific ReportsVol. 16(1)
Instituto de Biologia Experimental e Tecnológica (PT), Universidade Nova de Lisboa (PT), BOKU University (AT)
Austrian Science Fund, Ministério da Educação, Fundação para a Ciência e a Tecnologia
Openalex Percentile: Top 93%
Viral Infectious Diseases and Gene Expression in Insects
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