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Jun 7

Research Bit: Single cell RNA-Seq analysis of Cortical pathology in C9orf72 disease

Research Bits
The Packard Center welcomed Loyal Goff from Johns Hopkins University to our recent Investigator's Meeting.

Meeting Date: 7 June 2019

Presenter: Loyal A. Goff

Talk Title: Single cell RNA-Seq analysis of Cortical pathology in C9orf72 disease


What was the question being asked?

RNA-sequencing is a breakthrough technique used to study one of the main types of molecules in cells: RNA. RNA contains a type of code that helps cells translate genetic information from DNA into the enzymes and other proteins necessary for cellular function. In the context of ALS research, RNA-sequencing allows researchers to identify RNAs in degenerating neurons that are linked to cell death and disease progression. Once these changes are identified, therapies can be developed to prevent those changes early in the disease.

Why is this important for ALS research?

Researchers in the Goff Lab at Johns Hopkins are using RNA-sequencing to investigate if individual cells from the brains of ALS patients and unaffected individuals can be studied to pinpoint changes that drive disease progression in degenerating cells.

What was the take-home message?

RNA sequencing on single cells shows promise to identify differences between healthy and dying neurons. However, in order to better understand these differences and determine how important they are in ALS, many more samples will need to be sequenced.

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

This approach to understanding the disease is a powerful tool that can provide a large amount of data in a relatively short amount of time. Continuing work with RNA-sequencing will allow for more rapid research in the field of ALS.


Ben Zaepfel
Ph.D. Candidate | Rothstein Lab
Biochemistry, Cellular and Molecular Biology Ph.D. Program

The Johns Hopkins School of Medicine

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