Posted by Friends of FSH Research on Dec 10, 2024
Report by Dr. Kyle Siebenthall
See also TF-Scan Pilot Study
Talus Bio’s TF-Scan platform uses next-generation proteomics to discover drugs for proteins that regulate our genome, including transcription factors such as DUX4. In this pilot project, we used TF-Scan on human muscle cells (myoblasts) expressing DUX4 to verify our ability to find candidate drug-like molecules for DUX4, which is the first step toward discovering targeted small molecule inhibitors of DUX4 function. We observed robust increases in DUX4 activity when the protein is expressed, paired with increased abundance of known DUX4 targets like ZSCAN4. This project demonstrates that TF-Scan can be used for DUX4 drug discovery– as well as for other FSHD-relevant targets that may be discovered in the future– setting the stage for large-scale discovery efforts against DUX4 towards therapeutic development for patients with FSHD.
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