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Open access Oct 2026

Engineered Chimeric Proteins Functionally Replace ALS-causing Endogenous FUS and TDP-43 to Improve Neuronal Health

Mislocalization and aggregation of the RNA-binding proteins (RBPs) FUS and TDP-43 are pathological hallmarks of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), contributing to combined loss- and gain-of-function toxicity. Here, we present a protein engineering strategy that appends optimized anti...

Miyuki Hayashi, Brigid K. Jensen, A. Girdhar et al. · 0 citations
Open access Sep 2026

A Human Neuronal Cell Model of Endogenous TDP-43 A315T Reveals Altered Protein Dynamics and Disease-Relevant Cellular Dysfunction

A physiologically relevant human model is established that separates early TDP-43 toxic gain-of-function from basal loss-of-function from basal loss-of-function while providing a robust platform for investigating TDP-43 biology and accelerating therapeutic discovery in ALS.

M. Cicardi, Sara Antonini, Ivette Martorell Serra et al. · 0 citations

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