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Sep 11
2020

Research Bit: Targeting RAN proteins and the PKR pathway improves disease in C9-BAC ALS/FTD mice

Research Bits
The Packard Center welcomed Laura Ranum from the University of Florida to a recent Investigator's meeting

Meeting Date: September 11, 2020

Presenter: Laura Ranum

Talk Title: Targeting RAN proteins and the PKR pathway improves disease in C9-BAC ALS/FTD mice

What was the question being asked?

The Ranum lab seeks to understand how repeat proteins produced as a result of the C9orf72 mutation contribute to disease. They also hope to identify therapeutic strategies that reduce levels of these toxic proteins.

Why is this important for ALS research?

Repeat proteins produced as a result of the C9orf72 mutation in ALS/FTD have been shown to be highly toxic in multiple model systems. Thus, understanding how they are produced and exert toxicity is likely to lead to novel therapeutic strategies for the treatment of C9orf72 mediated neurodegeneration.

What was the take-home message?

Reducing levels of toxic C9orf72 repeat proteins in a mouse model of C9orf72 ALS/FTD mitigated multiple disease associated phenotypes.

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

The results of the studies being conducted within the Ranum lab suggest that strategies aimed at reducing levels of toxic C9orf72 repeat proteins may prove beneficial for C9orf72 ALS/FTD patients.

 

Prepared by:

Alyssa Coyne, PhD
Rothstein Lab
Johns Hopkins University

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