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May 21

Research Bit: Therapeutic strategies focusing on axonal health and neuromuscular junctions

Research Bits
The Packard Center welcomed Ludo Van Den Bosch, PhD from the VIB-KU Leuven Center for Brain & Disease Research to a recent Investigator's meeting.

Meeting Date: May 21, 2021

Presenter: Ludo Van Den Bosch

Talk Title: Therapeutic strategies focusing on axonal health and neuromuscular junctions


What was the question being asked?

Impaired transport along neuronal processes has long been regarded as a pathomechanism underlying neurogenerative diseases including ALS. Using patient derived neurons, the Van Den Bosch lab seeks to identify therapeutic approaches that can restore proper transport along these neuronal “highways”.

Why is this important for ALS research?

While altered transport along neuronal “highways” has been observed in ALS models for a number of years, few studies have evaluated the mechanism by which these disruptions occur. The Van Den Bosch lab has begun to unravel to neuronal basis of highway transport dysfunction using patient derived neuronal cultures. In turn, this work is poised to identify therapeutic targets that may mitigate neuronal dysfunction.

What was the take-home message?

Modifications to the microtubule highways ensure they are able to properly function. Using multiple patient derived neuronal models of familial ALS, the Van Den Bosch lab has shown that pharmacologically impairing the function of a protein that modifies these “highways” can restore proper transport. Moreover, they have found that manipulating the function of this protein can additionally restore the localization of TDP-43, an RNA binding protein commonly mislocalized in multiple neurodegenerative diseases including ALS.

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

Recent work in the Van Den Bosch lab has been exploring the potential of therapeutic targets to restore transport along neuronal highways and reverse TDP-43 pathology. These cellular alterations are shared amongst multiple neuropathies and neurodegenerative diseases. As a result, therapeutically targeting this neuronal transport pathway may prove beneficial for ALS as well as other related diseases.

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