Autor: Sara Emad El-Agamy, Francesca Mattedi and Pietro Fratta
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Cryptic Splicing in ALS: From Driving Disease Progression to Unlocking Novel Therapeutics
TDP-43 is an RNA-binding protein that regulates multiple aspects of RNA processing, and its mislocalization from the nucleus to the cytoplasm is a defining feature of amyotrophic lateral sclerosis (ALS). While both loss- and gain-of-function mechanisms…
