Article

Small molecule targeting r(UGGAA) <sub>n</sub> disrupts RNA foci and alleviates disease phenotype in <i>Drosophila</i> model

2020-10-05

Abstract excerpt

Synthetic small molecules modulating RNA structure and function have therapeutic potential for RNA diseases. Here we report our discovery that naphthyridine carbamate dimer (NCD) targets disease-causing r(UGGAA) n repeat RNAs in spinocerebellar ataxia type 31 (SCA31). Structural analysis of the NCD-UGGAA/UGGAA complex by nuclear magnetic resonance (NMR) spectroscopy clarified the mode of binding that recognizes f...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 7ed3a259-89cb-5457-a62c-a5112c192a37
DOI: 10.1101/2020.10.05.323261

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Small molecule targeting r(UGGAA) <sub>n</sub> disrupts RNA foci and alleviates disease phenotype in <i>Drosophila</i> modelDOI 10.1101/2020.10.05.323261Read

Small molecule targeting r(UGGAA)n disrupts RNA foci and alleviates disease phenotype in Drosophila model.Semantic proximity Read

Drosophila melanogaster As a Model Organism to Study RNA Toxicity of Repeat Expansion-Associated Neurodegenerative and Neuromuscular DiseasesSemantic proximity Read

Molecular Mechanisms and Future Therapeutics for Spinocerebellar Ataxia Type 31 (SCA31).Semantic proximity Read

Possibilities and challenges of small molecule organic compounds for the treatment of repeat diseasesSemantic proximity Read

Structures of RNA repeats associated with neurological diseasesSemantic proximity Read

A Toxic RNA Catalyzes the Cellular Synthesis of Its Own Inhibitor, Shunting It to Endogenous Decay PathwaysSemantic proximity Read

A small molecule that binds an RNA repeat expansion stimulates its decay via the exosome complexSemantic proximity Read

<i>C9orf72</i> repeat expansions cause neurodegeneration in <i>Drosophila</i> through arginine-rich proteinsSemantic proximity Read

Small molecule targeting of RNA structures in neurological disordersSemantic proximity Read

Structure and Dynamics of RNA Repeat Expansions That Cause Huntington's Disease and Myotonic Dystrophy Type 1.Semantic proximity Read

Interprotomer communication and functional asymmetry in H/ACA snoRNPs.Semantic proximity Read

Targeting Toxic RNAs that Cause Myotonic Dystrophy Type 1 (DM1) with a Bisamidinium InhibitorSemantic proximity Read