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Updated: Dec 31, 2025

Production of Adeno-Associated Virus Vectors in Cell Stacks for Preclinical Studies in Large Animal Models
Published on: June 30, 2021
Les vecteurs AAV pour le transfert de gène in vivo ou comment un petit virus devient grand
Rachel Millet1, Axel Rossi1, Aurélie Ploquin1
1CIRI Inserm U1111, CNRS UMR5308, équipe NucléoVir, 46, allée d'Italie, 69007 Lyon, France, École normale supérieure de Lyon, 46, allée d'Italie, 69007 Lyon, France, Université Lyon-I, 69622 Villeurbanne, France.
Abstract:
Among the variety of viral vectors, those derived from the human parvovirus Adeno-Associated Virus (AAV) have emerged as a very efficient tool for in vivo gene transfer into a variety of tissues and animal species during the two last decades. The relative simplicity of the organization of the AAV genome and the non-pathogenic property of the parental AAV has greatly contributed to the use of this viral vector among the gene transfer community. However, the limited knowledge of the wild type (wt) virus compared to other viral vectors has required considerable efforts to gain insight into wt AAV biology in order to improve the AAV vector system for therapy. This review will summarize the most important features of both wt and recombinant AAV to show how the increased understanding of the biology of the virus has enabled AAV vectors to lead the in vivo gene transfer field.
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