Dr Adam Mol
Adam is a Marie Skłdowska-Curie TRAIN@ED fellow working to create and adapt mammalian synthetic biology techniques and approaches to unmet needs in basic biology and medicine. In his endeavour, he adds more complexity to synthetic circuit design, such as producing more effective feedback control for therapeutic applications. Adam is also an expert designing inducible RNA-based systems to control cellular processes in mammalian cells. The goal of his project is to develop RNA-based regulators, which will provide a novel and appealing therapeutic approach to gene therapy.
In Novel RNA-based tools for cell and gene medicines project is 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 huge number of these targets are transcription factors, and modulating them is today one of the most difficult issues in cancer research, requiring innovation and the development of novel technologies. As a result, the goal of this project is to see if single-stranded RNA aptamers may be used to repress transcriptional regulators.
Adam is also designing expression cassettes capable of producing efficient and sub-toxic amounts of a therapeutic transgene for Rett Syndrome in collaboration with Stuart Cobb’s group. Rett syndrome (RTT) is an X-linked neurological illness caused mostly by methyl-CpG-binding protein 2 (MECP2) gene mutations. As a potential gene therapy for RTT, the goal of this effort is to deliver healthy copies of the MECP2 gene to compensate for the defective ones. The heterogeneity among cells caused by varied levels of infection is one of the obstacles of employing viral vectors to deliver therapeutic transgenes. Because excessive levels of MECP2 can be hazardous, it’s critical to develop a way to reduce MECP2 overexpression in severely infected cells as a potential RTT gene therapy.
This TRAIN@Ed project has received funding from the European Union’s Horizon 2020 research and innovation programme under the Marie Skłodowska-Curie grant agreement No 801215 and UK Mammalian Synthetic Biology Research Centre BBSRC BB/M018040/1