Exploring the Potential of Small Molecule-Based Therapeutic Approaches for Targeting Trinucleotide Repeat Disorders
Author:
Publisher
Springer Science and Business Media LLC
Subject
Neuroscience (miscellaneous),Cellular and Molecular Neuroscience,Neurology
Link
http://link.springer.com/content/pdf/10.1007/s12035-019-01724-4.pdf
Reference219 articles.
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3. Lopez Castel A, Cleary JD, Pearson CE (2010) Repeat instability as the basis for human diseases and as a potential target for therapy. Nat Rev Mol Cell Biol 11(3):165–170. https://doi.org/10.1038/nrm2854
4. 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
5. Marelli C, Maschat F (2016) The P42 peptide and peptide-based therapies for Huntington's disease. Orphanet J Rare Dis 11:24. https://doi.org/10.1186/s13023-016-0405-3
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