| Autor: |
Paco Pino, Coralie Croissant, Joshua Armitano, Bertrand Lazuech, Danijel Švec, Cyril Pugin, Anaïs Guesdon, Louise Bryan, Antonio Castro, Léa Neuhaus, Giulia Fonti, Jacopo Martinis, Maria Wurm, Florian M. Wurm |
| Rok vydání: |
2023 |
| DOI: |
10.22541/au.167424853.33255289/v1 |
| Popis: |
Viral vectors for gene therapy, such as recombinant Adeno-Associated Viruses (rAAV), are produced in Human Embryonic Kidney (HEK) 293 cells. However, the presence of the SV40 T-antigen-encoding CDS SV40GP6 and SV40GP7 in the HEK293T genome raises safety issues when these cells are used in manufacturing for clinical purposes. We developed a new T-antigen-negative HEK cell line from ExcellGene’s proprietary HEKExpress®, using the CRISPR-Cas9 strategy. We obtained a high number of clonally-derived cell populations and all of them were demonstrated T-antigen negative. Stability study and AAV production evaluation showed that the deletion of the T-antigen-encoding locus did not impact neither cell growth nor viability nor productivity. The resulting CMC-compliant cell line, named HEKzeroT®, is able to produce high AAV titers, from small to large scale. |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
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