Seminar: Pietro Fratta

Pietro Fratta is Professor of Cellular and Molecular Neuroscience at the UCL Queen Square Institute of Neurology and the Francis Crick Institute. He previously worked at King’s College London, University of Southern California and San Raffaele Scientific Research Institute.
His laboratory combines patient tissue, iPSCs and mouse models to understand molecular disease mechanisms underlying ALS and neurodegeneration. Over the last five years, his laboratory has developed a novel gene therapy approach to limit therapeutic expression to diseased cells, uncovered the role of UNC13A in ALS and developed a splice-switching ASO therapeutics now in clinical trial with Trace Neuroscience, and identified novel biomarkers for ALS.
He is also a Consultant Neurologist at the National Hospital for Neurology and Neurosurgery in London, where he established National Kennedy’s Disease clinic and an MND Genetics clinic.

You can read the following articles to see some of Prof. Pietro Fratta's work:

Nature Neuroscience:

TDP-43 loss induces cryptic polyadenylation in ALS/FTD

TDP-43-dependent mis-splicing of KCNQ2 triggers intrinsic neuronal hyperexcitability in ALS/FTD

Science Direct:

Seeded aggregation of TDP-43 induces its loss of function and reveals early pathological signatures

Science:

Creation of de novo cryptic splicing for ALS and FTD precision medicine

Nature:

TDP-43 loss and ALS-risk SNPs drive mis-splicing and depletion of UNC13A