MicroRNA-regulated viral vectors for gene therapy

Figure 1 Principle of microRNA-mediated suppression of transgene expression and viral replication. After transduction/infection of a cell, vector DNA/viral DNA is transcribed in the nucleus (for lentiviral vectors integrated into host DNA). The transcript/viral RNA containing artificial miR-TS (red boxes) is transported into the cytoplasm. If corresponding microRNA is expressed (A), it binds to miR-TS and the target RNA is endonucleolytically cleaved and degraded. If certain microRNAs are not expressed (B), the RNA is translated into protein. The protein shown in the three-dimensional structure is dimeric S100A1[180]. AGO: Argonaute; miRISC: MicroRNA-induced silencing complex.