Synthetic transcriptional activators (syn-TFs): a new therapeutic platform for the treatment of Friedrich's Ataxia (FRDA)

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One of the holy grails of genetic medicine is to develop therapies which can correct aberrant gene expression. This is particularly challenging with genetic diseases such as Friedrich's Ataxia (FRDA). FRDA arises when repeating GAA-TTC sequences of DNA build up in the frataxin gene (FXN), which results in a significant reduction in the production of frataxin. The overall objective of this studentship is to develop a new generation of chimeric molecules which can activate FXN transcription. The studentship will involve a blend of synthetic chemistry, chemical biological probe design, and compound optimization guided by deep-sequencing analysis. This project directly aligns with the EPSRC's Chemical Biology & Biological Chemistry research area in the Healthcare Technologies Theme.