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Updated: Jun 27, 2025

Author Spotlight: Advancing Gene Therapy Research with High-Titer Adeno-Associated Virus Vector Production
Published on: May 3, 2024
Unravelling the essential elements for recombinant adeno-associated virus (rAAV) production in animal cell-based
David Catalán-Tatjer1, Konstantina Tzimou1, Lars K Nielsen2
1The Novo Nordisk Foundation Center for Biosustainability, Technical University of Denmark, Denmark.
Abstract:
Recombinant adeno-associated viruses (rAAVs) stand at the forefront of gene therapy applications, holding immense significance for their safe and efficient gene delivery capabilities. The constantly increasing and unmet demand for rAAVs underscores the need for a more comprehensive understanding of AAV biology and its impact on rAAV production. In this literature review, we delved into AAV biology and rAAV manufacturing bioprocesses, unravelling the functions and essentiality of proteins involved in rAAV production. We discuss the interconnections between these proteins and how they affect the choice of rAAV production platform. By addressing existing inconsistencies, literature gaps and limitations, this review aims to define a minimal set of genes that are essential for rAAV production, providing the potential to advance rAAV biomanufacturing, with a focus on minimizing the genetic load within rAAV-producing cells.

