SEP 11, 2020 6:39 PM PDT

Novel Therapeutic for ALS

WRITTEN BY: Nouran Amin

The TDP-43 protein is a well-known hallmark of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). A pre-clinical study found that an investigational drug may target the particular hallmark protein.

"A common genetic cause of ALS and FTD is a repeat expansion in the C9orf72 gene, which somehow leads to TDP-43 aggregation in degenerating neurons, but what remained unclear until now was how those two were connected," said co-senior author James Shorter, PhD, a professor of Biochemistry and Biophysics in the Perelman School of Medicine at the University of Pennsylvania. "We found that TDP-43 aggregates much more rapidly if these toxic poly(GR) proteins are around, suggesting a direct link between the mutation, poly(GR), and TDP-43."

Learn more about amyotrophic lateral sclerosis (ALS):

Findings were published in Science Translational Medicine.

"This finding presents an exciting potential therapeutic target to treat these debilitating diseases by lowering poly(GR) levels," added Hana Odeh, PhD, a post-doctoral fellow in the Shorter lab and co-first author.

Source: Science Daily

About the Author
  • Nouran is a scientist, educator, and life-long learner with a passion for making science more communicable. When not busy in the lab isolating blood macrophages, she enjoys writing on various STEM topics.
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