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Departamento de Biología

Seminario del 30 de abril de 2021

Ponente: Óscar Fernández-Capetillo (CNIO)

Título: Mechanisms, mouse models and drug development: A cancer researcher´s approach to amyotrophic lateral sclerosis

Día: Viernes 30 de abril, a las 12h. Reunión en Teams siguiendo el siguiente enlace:

ALS is a progressive and fatal neurodegenerative disease which currently lacks a cure. Studies revealed in the previous decade identified mutations in a gene named C9ORF72 as the most frequent one in ALS patients. The biology behind how mutations in C9ORF72 lead to ALS is fascinating, and most studies converge to indicate that this is due to the production of some toxic arginine-rich peptides which kill motoneurons. Still, many important questions remain to be addressed. Why are these arginine-rich peptides toxic? Can we rescue their toxicity? Can we develop preclinical mouse models that express these peptides and that could be used for the development of new therapies? And, finally, can we identify such therapies? Capitalizing in our experience in the development of mouse models and drug development in the context of cancer, during the last 5 years our group has been trying to provide answers to these questions, with the ultimate goal of trying to contribute to the search of a cure for ALS. Our ideas and progress in this topic will be discussed.

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