The great potential of gene therapy is currently limited by two major challenges: tissue specific gene delivery and regulation of gene expression, either dependent on cell-specific properties or intentionally independent of the cellular context. We followed two synergistic tracks to address these problems.
One, we have developed a novel method for miRNA based gene expression tuning in mammalian cells, allowing the fine-tuning of gene expression based on synthetic miRNAs, as well as the cell specific on- and off-targeting based on endogenous miRNAs. We show that this method is functional in vivo and in vitro and prove the high potential of all three miRNA-based regulation approaches.
Two, we have developed a standardized and fast approach towards the creation of AAV-based gene delivery vectors. We have achieved exceptionally selective tissue-specific targeting in vitro and in vivo with hepatocyte specific delivery vectors.
We are happy to provide the synthetic biology community with two high impact innovations which will fuel the improvement of tissue specific gene therapy approaches and other medical applications of synthetic biology.
"
