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Aggregated Proteins

An abnormal clumping of proteins in neurons is a common feature of Alzheimer’s, Parkinson’s and other neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS). Researchers now have multiple lines of evidence suggesting that these protein clumps are toxic to cells.

Insoluble aggregates of the SOD1 protein typical of familial ALS, for example, are found

Packard researchers found that aggregates of SOD1 protein appear early on and accumulate as the disease progresses, suggesting that aggregation may be an early event in ALS’s pathology. SOD1 protein clumps are also found in in the motor neurons of model ALS mice, just before or at the same time that ALS symptoms begin.

Recently, aggregates of two other proteins, TDP-43 and FUS, were isolated in ALS patients – mostly those with the familial disease, though some with sporadic. The fact that they appear in both types of patients is important. More studies will show if the proteins are part of what triggers ALS onset or progression. The appearance of these aggregates provides, in principle, the possibility of a prime drug target – one that lets us stop or slow the disease early on.

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Johns Hopkins University
Motor neurons can only work properly if the cell’s proteins can get to the right place at the right time. Thomas Lloyd uses the fruit fly Drosophila melanogaster to study how proteins are shuttled between the cell body and the synapse, as interruptions in this process have been linked to ALS. 
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University of Michigan
Sami Barmada wants to answer a very basic question about ALS: why motor neurons? Of all the different types of neurons in the body (and scientists estimate there are probably several hundred), it’s only motor neurons that are affected in ALS. Knowing why this is, Barmada believes, could be the key to developing new potential treatments that could prevent the deterioration and death of motor neurons. 
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