The key to successful gene therapy is integration of tissue specificity and fine-tuned target gene expression. The iGEM Team Heidelberg 2010 unlocks the world of synthetic microRNAs, since focusing solely on DNA has often been inconvenient for medical purposes. We engineered a toolkit for standardized measurements of interactions between artificial miRNAs and their binding sites. From this data we were able to compute an in silico model integrating binding site properties and knockdown percentages. Thus, the expression level of any gene of choice could be arbitrarily adjusted by employing the corresponding binding site design. To produce tissue specific miRNA gene shuttles, we developed an evolution-based method for synthesis of new adeno associated viruses. This enabled us to overcome the natural limitations of virus selectivity. In the future, miBricks could be applied for treatment of diseases like Diabetes and Hemophilia, opening the doors to new Synthetic Biology based medical approaches.
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