This is the Medscape Neurology Minute. I'm Dr Alan Jacobs.
Researchers at the University of Toronto have been investigating a new and potentially very important way in which amyotrophic lateral sclerosis (ALS) kills motors neurons. They focus on an RNA binding protein called fused in sarcoma (FUS), which is known to accumulate in ALS motor neurons.[1]
They've shown that the FUS protein has the unusual ability to morph between liquid and gel forms. The gel form of FUS allows it to collect other cellular components and transport them in compact, concentrated forms to distant edges of the neurons where FUS then melts back to its liquid form, allowing protein synthesis to occur.
Now, these researchers have found that mutations in FUS cause it to form very dense gels that resist remelting and kill the neuron by preventing protein synthesis. Their next goal is to find ways to prevent the solidification of the gel or to reverse the hardening process. They see this therapeutic strategy as also very likely to be applicable to ALS and frontotemporal dementia associated with mutations in other RNA binding proteins.
This has been the Medscape Neurology Minute. I'm Dr Alan Jacobs.
Medscape Neurology © 2016 WebMD, LLC
Any views expressed above are the author's own and do not necessarily reflect the views of WebMD or Medscape.
Cite this: A Potential Therapeutic Target in ALS and Dementia - Medscape - Mar 15, 2016.
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