Single-gene disorders originate in the absence or loss of function of a protein due to a genetic mutation. Gene therapy is a promising therapeutic approach that delivers a normal version of the gene to affected cells to compensate for its missing or defective counterpart. It often employs viral vectors, such as recombinant Adeno Associated Viruses (rAAVs), to insert the genes. The insect cell line Sf9 provides a suitable host for virus production. Sf9 cells are cultured in suspension, and hence working volumes can be adapted to changing needs during process development and manufacturing much more easily than for adherent cell cultures. In this study, researchers at Généthon® developed a scale-down model for rAAV viral vector production in Sf9 cells using an Eppendorf DASbox® Mini Bioreactor System. Parallel experimentation in small working volumes allowed timeand cost-efficient evaluation of process performance.
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Original Publication Date: 03/01/2016