Novel aptamer tools for Synthetic Biology and the control in cell and gene therapies

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Aptamers are rapidly developing oligonucleotide tools that consist of single stranded DNA or RNA molecules that are capable of binding nearly any ligand of choice with high affinity and specificity due to their specific three-dimensional structures. Aptamers are rapidly maturing from a simple research tool into a major technology with huge commercial potential. Aptamers provide a promising alternative to conventional therapeutic approaches as they elicit no immunogenicity or toxicity and can be easily modified to increase their stability in biological environments. Moreover, aptamers can be produced quickly and relatively inexpensively. These remarkable properties are reflected by the first aptamer-based therapeutic agent (Macugen) clinically approved as a drug for treating a form of macular degeneration. In recent years, more and more researchers and pharmaceutical companies are realizing the enormous potential of aptamers and aptamer-based products will enter the market in the near future 1,2,3. In this PhD we will investigate the potential of aptamers to be developed for controlling cell processes and targeting biomarkers that are considered 'undruggable'; that cannot be targeted pharmacologically. A large percentage of these targets fall under the category of transcription factors (TFs) and their modulation is now one of key challenges in cancer research that requires innovation and the development of new technologies. Therefore, we will explore whether we could suppress transcriptional regulators via a single-stranded RNA aptamers. We will also develop aptamer based synthetic gene regulatory circuits for control of cellular processes.