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Oct 15
2021

Research Bit: From disease mechanisms towards therapy development in FUS-mediated proteinopathies

Research Bits
The Packard Center welcomed Sandrine Da Cruz from VIB-KU Leuven Center to a recent Investigator's meeting.

Date: October 15, 2021

Presenter: Sandrine Da Cruz, PhD

Talk Title: From disease mechanisms towards therapy development in FUS-mediated proteinopathies

What was the question being asked?

The Da Cruz lab is exploring how the spread of RNA binding proteins, including FUS and TDP-43, contributes to the progression of ALS and FTD. 

Why is this important for ALS research?

The separation and aggregation of these disease-causing proteins contributes to neurodegeneration. The Da Cruz lab aims to identify therapeutic strategies to target neuromuscular junction to rescue motor neuron cell bodies and improve muscle innovation.

 What was the take-home message?

Motor neurons in the central nervous system signal to muscles through neuromuscular junctions, which are critical for motor neurons to induce contractions of muscles that regulate basic bodily functions. The Da Cruz lab used multiple models to evaluate the impact of aggregates on neuromuscular junctures.

How do you think the results of this study might impact future approaches to the treatment of ALS?

This work aims to validate compounds that target pathways (i.e., synaptic transmission and clustering of receptors in the muscle) to find therapeutic approaches to improve quality of life and sustained function for patients with ALS and FTD.

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