16 research outputs found
Small molecule targeting r(UGGAA)n disrupts RNA foci and alleviates disease phenotype in Drosophila model
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Ligand-RNA interaction assay based on size-selective fluorescence core-shell nanocomposite
No full textNucleobase-Modified Triplex-Forming Peptide Nucleic Acids for Sequence-Specific Recognition of Double-Stranded RNA
No full textBecause of the important roles noncoding RNAs play in gene expression, their sequence-specific recognition is important for both fundamental science and the pharmaceutical industry. However, most noncoding RNAs fold in complex helical structures that are challenging problems for molecular recognition. Herein, we describe a method for sequence-specific recognition of double-stranded RNA using peptide nucleic acids (PNAs) that form triple helices in the major grove of RNA under physiologically relevant conditions. We also outline methods for solid-phase conjugation of PNA with cell-penetrating peptides and fluorescent dyes. Protocols for PNA preparation and binding studies using isothermal titration calorimetry are described in detail
Design of a bioactive small molecule that targets r(AUUCU) repeats in spinocerebellar ataxia 10
No full textNon-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1
No full textAffinity capillary electrophoresis for identification of active drug candidates in myotonic dystrophy type 1
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