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.
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The iGEM idea
iGEM (international Genetically Engineered Machines Competition) is an international competition in synthetic biology, hosted by the MIT in Boston. The aim of this competition is to answer a basic question once posted by the director of iGEM, Randy Rettberg, as follows: "Can simple biological systems be built from standard, interchangeable parts and operated in living cells? Or is biology just too complicated to be engineered in this way?"
International student teams participating in the iGEM compete to answer this fundamental question by engineering biological systems with a proper function. More than 100 interdisciplinary student teams from all over the world, mainly consisting of undergraduate students in biology, biochemistry, engineering, informatics and mathematics, carry out different projects during the Summer to follow this approach.
Projects involved in iGEM reach from medical applications, such as genetically modified bacteria used in cancer-treatment to environmental and manufacturing projects, which allow the construction of a dynamic, watch-like counter consisting of living cells. In contrast to classical genetic engineering where only one gene is transferred from organism A to organism B, synthetic biology advances into the construction of new systems as a whole with totally new emerging properties. Therefore, each iGEM-Teams gets access to a gene-Database called "registry", where hundreds of different genetic parts with characterized functions are available in a “plug-and-play”–like manner. These parts can be simply stuck together to create new functional systems. The rising number of iGEM-Teams over the last years as well as the upcoming public interest in iGEM as well as in the iGEM-Teams’ projects and synthetic biology in general shows that synthetic biology will demonstrate an essential contribution to understand the functional way of life and have an enormous impact on many different fields of both scientific reseach and every-day life.