Integrated computational and experimental approaches to drug repositioning for rare genetic disorders

Our lab develops and applies innovative technologies integrating experimental and computational approaches to study and engineer living organisms with the aim of advancing therapies for rare and common disorders. One of the main tools in our research is microfluidics, a technique that allows cell probing in real-time and precise measuring of individual cell response over time thus enabling quantitative observation and mathematical modelling of biological processes. The lab has exploited this platform to achieve precise temporal control of transcriptional and signalling pathways in yeast and mammalian cells. We are currently using it to gain novel understanding on how the stress response in mammalian cells is regulated and how it could be re-engineered to yield super-producer cell lines capable of speeding up the production of biological drugs and viral vectors for gene therapy while reducing costs. Our lab has also pioneered the concept of using transcriptional responses to drug treatments in cells and tissues to reposition drugs for the therapy of rare genetic disorders and common diseases. We are now extending this approach to the use of single-cell transcriptomics in cancer to identify drug combinations to prevent development of drug resistance. The "Total Award" amount indicated for this project represents the share of the funding of the Telethon Foundation for research by the Tigem institute from January 2022 until last budget year, calculated based on the size of the research group.