Graham is a co-inventor of synthetic transcription elongation factors (Syn-TEFs), a new class of TR-targeting compounds that reverse pathogenic gene expression. Syn-TEF1, which targets the pathogenic repeat expansion in Friedreich’s ataxia, is the most potent activator of frataxin expression reported to date. An analog of Syn-TEF1, developed by Design Therapeutics, is scheduled to enter clinical trials in the first half of 2022.
Graham is interested in building the next generation of TR-targeting compounds to reverse the effects of pathogenic repeat expansions.
We develop and deploy new tools to identify variations in TR DNA sequences in human genomes.
Our long-term goal is to elucidate the function of TR sequences in the human genome.