Suppression of toxic transgene expression by optimized artificial miRNAs increases AAV vector yields in HEK-293 cells.

Normalized luminescence revealed that the expression of all five SplashRNA-based, miR-F expressed miRNAs led to highly significant and efficient silencing of FLuc, with knockdown efficiencies of 94%–96%, as compared with the miRNA-free control ( Figure 1 C). amiR-001 and amiR-002 as well as cel-miR39 transfection also resulted in significant silencing of FLuc, however, at a lower potency, with remaining FLuc activities of 11%–60%.

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