Elena Cattaneo is the Director of the Laboratory of Stem Cell Biology and Pharmacology of Neurodegenerative Diseases at the Department of Biosciences and co-founder and Director of UniStem, the Centre for Stem Cell Research at the University of Milan. Since 2015, her laboratory has been hosted by the National Institute of Molecular Genetics (INGM) under a formal partnership.

Her laboratory investigates the molecular pathophysiology and treatment of Huntington’s Disease (HD). Using precise cellular and animal models, they study the mechanisms driving striatal neurodegeneration. They employ embryonic stem cell technologies to generate medium-sized spiny neurons—the population most affected in HD—and develop cellular platforms to explore disease processes. Studies of fetal striatal development in vivo by single-cell RNA sequencing aim to improve differentiation of striatal neurons in vitro, which are then tested in transplantation paradigms. The laboratory also examines developmental components of HD, as well as roles of cholesterol metabolism and synaptic signaling pathways. Another line of research addresses the function and evolution of the huntingtin gene, seeking molecular targets for therapy.

Her work has been supported by the Huntington’s Disease Society of America, the Hereditary Disease Foundation, the CHDI Foundation, the European Commission (FP7, H2020), the Italian Ministry of Research (PRIN), Fondazione Telethon, and others. She coordinated three EU-funded consortia—NeuroStemCell (2008–2012), NeuroStemCell-Repair (2013–2017), and NeuroStemCell-Reconstruct (2019–2024)—and leads an Italian network on stem cell transplantation for HD. In 2018, she received an Advanced ERC Grant, and in 2024, an ERC Synergy Grant with three colleagues, scheduled to begin in November 2025. Under a CHDI program (2016–2024), her team tested whether HD shows transcriptional changes already in fetal development. Using single-cell RNA-seq of mouse and human cortex and striatum—including one rare human HD fetal sample—they generated extensive datasets, although spatial transcriptomics were beyond their reach at that time.

Elena Cattaneo has published over 200 articles in peer-reviewed journals including Science, Nature, and The Journal of Neuroscience, with an H-index of 68. She has taught courses such as “Stem Cells in Pharmacology and Regenerative Medicine,” and, since her 2013 appointment as Senator for Life, she continues to lecture, though she no longer runs formal courses. She has supervised over 30 PhD and 110 Master’s students and delivered more than 400 invited talks worldwide.

In 2006, she co-founded UniStem, dedicated to stem cell research and public outreach. The centre organizes workshops, lectures, and a large annual event for high-school students, now reaching 25,000 participants across 90 European universities.

In 2013, President Giorgio Napolitano appointed her Senator for Life in recognition of her scientific and social contributions. She remains the youngest and only the third woman in Italian history to hold this role. In the Senate, she focuses on science, innovation, freedom of research, human rights, and education.

Talk title: Brain Cholesterol Matters: A 25-Year Journey Toward Nanomedicine-Based Therapies for Huntington’s Disease