Small Molecule Screening Discovers Compounds that Reduce FMRpolyG Protein Aggregates and Splicing Defect Toxicity in Fragile X-Associated Tremor/Ataxia Syndrome
Author:
Publisher
Springer Science and Business Media LLC
Subject
Neuroscience (miscellaneous),Cellular and Molecular Neuroscience,Neurology
Link
https://link.springer.com/content/pdf/10.1007/s12035-021-02697-z.pdf
Reference59 articles.
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3. Verma AK, Khan E, Bhagwat SR, Kumar A (2019) Exploring the potential of small molecule-based therapeutic approaches for targeting trinucleotide repeat disorders. Mol Neurobiol. https://doi.org/10.1007/s12035-019-01724-4
4. Warner KD, Hajdin CE, Weeks KM (2018) Principles for targeting RNA with drug-like small molecules. Nat Rev Drug Discovery 17(8):547–558. https://doi.org/10.1038/nrd.2018.93
5. Krzyzosiak WJ, Sobczak K, Wojciechowska M, Fiszer A, Mykowska A, Kozlowski P (2012) Triplet repeat RNA structure and its role as pathogenic agent and therapeutic target. Nucleic Acids Res 40(1):11–26. https://doi.org/10.1093/nar/gkr729
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