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Small Molecule Targeting TDP-43's RNA Recognition Motifs Reduces Locomotor Defects in a Model of Amyotrophic Lateral Sclerosis (ALS).

ACS Chem Biol 2019 09 27;14(9):2006-2013. Epub 2019 Aug 27.

Department of Pharmacology, College of Medicine , University of Arizona , Tucson , Arizona 85724 , United States.

RNA dysregulation likely contributes to disease pathogenesis of amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. A pathological form of the transactive response (TAR) DNA binding protein (TDP-43) binds to RNA in stress granules and forms membraneless, amyloid-like TDP-43 aggregates in the cytoplasm of ALS motor neurons. In this study, we hypothesized that by targeting the RNA recognition motif (RRM) domains of TDP-43 that confer a pathogenic interaction between TDP-43 and RNA, motor neuron toxicity could be reduced. Read More

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September 2019
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