Post by CeMM

🚀 #𝗙𝗿𝗼𝗻𝘁𝗥𝗼𝘄𝗦𝗰𝗶𝗲𝗻𝗰𝗲 𝗰𝗼𝗻𝘁𝗶𝗻𝘂𝗲𝘀 𝘄𝗶𝘁𝗵 𝗵𝗶𝗴𝗵𝗹𝗶𝗴𝗵𝘁𝘀 𝗳𝗿𝗼𝗺 𝗼𝘂𝗿 #𝗖𝗲𝗠𝗠𝗥𝗲𝘀𝗲𝗮𝗿𝗰𝗵𝗥𝗲𝗽𝗼𝗿𝘁𝟮𝟬𝟮𝟱! Today, we feature CeMM research groups working on disease model systems: 🧬 Acute myeloid leukemia (AML) is an aggressive cancer of white blood cells. In some cases, chromosomal rearrangements create fusions between two genes, leading to fusion oncoproteins that drive uncontrolled cell growth, a phenomenon particularly common in children with AML. The group of CeMM Adjunct PI Florian Grebien (Vetmeduni) studies the molecular mechanisms of leukemia development, with a particular focus on the cellular and molecular effects of these "oncofusions". In 2025, they reported two new potential therapeutic avenues for NUP98::KDM5A-driven AML. As Grebien noted during the group’s discussion at the 𝘛𝘩𝘦𝘢𝘵𝘦𝘳 𝘕𝘦𝘴𝘵𝘳𝘰𝘺𝘩𝘰𝘧 𝘏𝘢𝘮𝘢𝘬𝘰𝘮, “We want to find vulnerabilities that are very precise for a given type of cancer so that, in the future, we can develop more specific therapies with fewer side effects.” 👉 https://bit.ly/4dZ4V0Z 🧪 Dysregulated gene control is a major driver of cancer formation and drug resistance in leukemia. At St. Anna Children's Cancer Research Institute (CCRI), CeMM Adjunct PI Davide Seruggia’s group investigates gene regulation in pediatric leukemia using molecular and computational approaches. They studied collateral degradation in the SAGA transcriptional co-activator complex, which has been implicated in AML, and identified a promising alternative to traditional enzymatic inhibition: targeting its structural core rather than the cancer-driving protein itself. At the 𝘞𝘪𝘦𝘯𝘦𝘳 𝘒𝘰𝘯𝘻𝘦𝘳𝘵𝘩𝘢𝘶𝘴, the group emphasized the unique challenges of studying childhood cancers: “Most of what we learn in cancer research in adults does not apply to pediatric cancer,” said Seruggia, highlighting the importance of developing specific, safe therapies that eliminate cancer cells without affecting future growth, development, and fertility. 👉 https://bit.ly/4dUbn9q 🔬 The group of CeMM Adjunct PI Andreas Villunger (Medizinische Universität Innsbruck) studies cell death signaling in health and disease, including the crosstalk between the cell cycle and cell death machinery, which is relevant for cancer. One project took the group in an unexpected direction, leading them to a family of enzymes that regulate transfer RNA (tRNA) modification. Their work revealed how defects in thiolation can cause a rare disease. The same mutation had little effect in mice, opening avenues for future research to understand whether compensatory mechanisms prevent disease in this model. During their discussion at the 𝘛𝘩𝘦𝘢𝘵𝘦𝘳 𝘢𝘮 𝘚𝘱𝘪𝘵𝘵𝘦𝘭𝘣𝘦𝘳𝘨, Villunger noted that “most breakthrough discoveries were not made by predetermined, top-down research projects but by scientists with the freedom to be creative and pursue crazy ideas.” 👉 https://bit.ly/4sFDBbK 📸 Klaus Pichler / CeMM