Fratta Pietro

Dr. Fratta Pietro is Professor of Cellular and Molecular Neuroscience and a Consultant Neurologist, and his clinical and research interests center on motor neuron diseases (MND) and RNA biology. He has been leading an independent research team since 2015 in University College London, and since 2023 at the Francis Crick Institute. He is a Consultant Neurologist at the National Hospital for Neurology and Neurosurgery, where he established in 2015 the only Kennedy’s Disease-dedicated clinic in the UK and an MND Genetics clinic. He previously trained at UCL, King’s College London, San Raffaele Scientific Institute in Milan, and the University of Southern California. His laboratory uses patient derived tissue, iPS cells and mouse models to understand disease mechanisms. They are developing gene therapy approaches to target the sporadic ALS population, and they harness the longitudinal sample collections in their cohorts to develop biomarkers using patient tissue and biofluids. Over the last five years his laboratory has 1) discovered novel molecular consequences of TDP-43 dysfunction leading to the development of novel biomarkers; 2) identified TDP-43 targets that impact disease progression and devised therapeutic strategies to correct these, currently entering clinical trials; 3) developed precision medicine gene therapy technologies to specifically target the diseased cells in ALS.