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Monobac System–A Single Baculovirus for the Production of rAAV

Large-scale manufacturing of rAAV is a bottleneck for the development of genetic disease treatments. The baculovirus/Sf9 cell system underpins the first rAAV treatment approved by EMA and remains one of the most advanced platforms for rAAV manufacturing. Despite early successes, rAAV is still a comp...

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Detalles Bibliográficos
Autores principales: Galibert, Lionel, Jacob, Aurélien, Savy, Adrien, Dickx, Yohann, Bonnin, Delphine, Lecomte, Christophe, Rivollet, Lise, Sanatine, Peggy, Boutin Fontaine, Marjorie, Le Bec, Christine, Merten, Otto-Wilhelm
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8465638/
https://www.ncbi.nlm.nih.gov/pubmed/34576695
http://dx.doi.org/10.3390/microorganisms9091799
Descripción
Sumario:Large-scale manufacturing of rAAV is a bottleneck for the development of genetic disease treatments. The baculovirus/Sf9 cell system underpins the first rAAV treatment approved by EMA and remains one of the most advanced platforms for rAAV manufacturing. Despite early successes, rAAV is still a complex biomaterial to produce. Efficient production of the recombinant viral vector requires that AAV replicase and capsid genes be co-located with the recombinant AAV genome. Here, we present the Monobac system, a singular, modified baculovirus genome that contains all of these functions. To assess the relative yields between the dual baculovirus and Monobac systems, we prepared each system with a transgene encoding γSGC and evaluated vectors’ potency in vivo. Our results show that rAAV production using the Monobac system not only yields higher titers of rAAV vector but also a lower amount of DNA contamination from baculovirus.