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Hope for ALS Patients Comes from New Understanding of RNA Metabolism Misregulation

05:23 EDT 19 Mar 2018 | Genetic Engineering News

An international team led by neuroscientists at Université de Montréal reports the discovery of a basic molecular mechanism that better helps understand how Lou Gehrig's disease, or amyotrophic lateral sclerosis (ALS), works. Their finding may eventually lead to new treatments for the debilitating disease, which cripples people by removing the brain's ability to communicate with their muscles, eventually leading to paralysis and early death, according to the scientists. "It's a story of fundamental research about what happens normally in the body's cells and what changes in the context of ALS," said Jade-Emmanuelle Deshaies, a research associate in neurosciences at the UdeM Hospital Research Centre (CRCHUM) and lead author of the joint Canada-Israel study (“ TDP-43 regulates the alternative splicing of hnRNP A1 to yield an aggregation-prone variant in amyotrophic lateral sclerosis ”), published in  Brain . “The RNA binding proteins TDP-43 (encoded by  TARDBP ) and hnRNP A1 ...

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