CMCB Life Sciences Seminar: Prof. Pietro Fratta, University College London, The Francis Crick Institute, UK

Host: Jared Sterneckert (CRTD)

Title: "Cryptic splicing: from foe to friend in tackling ALS”

Abstract: The Fratta Lab uses patient derived tissue, iPS cells and mouse models to understand disease mechanisms in amyotrophic lateral sclerosis (ALS), with a focus on RNA metabolism.  Over the last five years the laboratory has 1) discovered novel molecular consequences of TDP-43 dysfunction leading to biomarkers of disease; 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.