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Feb 19
2021

Research Bit: Cell intrinsic discrepancy in human ALS motor neuron subtypes reveals a converged disease mechanism and target metabolites

Research Bits
The Packard Center welcomed Gabsang Lee from the Johns Hopkins University to a recent Investigator's meeting.

Date: February 19, 2021

Presenter: Gabsang Lee, PhD

Talk Title: Cell intrinsic discrepancy in human ALS motor neuron subtypes reveals a converged disease mechanism and target metabolites

What was the question being asked?

The neurodegenerative diseases ALS and FTD share common genetic causes yet manifest as two clinically distinct diseases. Further, while mutant genes are present in all cells of the body, the cell types affected in disease are distinct. The Lee seeks to address this critical outstanding question regarding what makes some cell types more susceptible to degeneration elicited by genetic mutations.

Why is this important for ALS research?

This work will provide insights into the cell biological differences underlying different neuronal subtypes. Further, understanding cell type specific pathophysiology can help to identify targeted therapeutic strategies.

What was the take-home message?

The Lee lab compared disease affected neuronal populations (spinal motor neurons) to those spared in ALS (ocular motor neurons). In doing so, they identified a subset of genes, namely those involved in lipid metabolism, whose expression was specifically altered in ALS spinal motor neurons.

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

Identification of ALS spinal motor neuron specific pathophysiology may provide insights into more targeted therapeutic approaches for cell type specific neurodegenerative disease.

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