Titlebook: RNA Therapeutics; Amanda L. Garner Book 2018 Springer International Publishing AG 2018 microRNAs.therapeutic approach.G-quadruplexes.viral

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1862-2461 teractions on the molecular level, structure-activity relationships, drug absorption, distribution, metabolism, elimination, toxicology and pharmacogenomics. In general, special volumes are edited by well known978-3-319-88546-9978-3-319-68091-0Series ISSN 1862-2461 Series E-ISSN 1862-247X

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Computational Tools for Design of Selective Small Molecules Targeting RNA: From Small Molecule Micre compounds. For example, lead compounds that target the r(CCUG) repeats expansions that cause myotonic dystrophy type 2 (DM2) were lead optimized by using structure-based design. Specifically, the compounds were developed to allow an in situ click chemistry approach in which a disease-affected cell

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Drugging Pre-mRNA Splicing,d under natural conditions. This approach has powerful therapeutic utility where mutation has caused certain splice variants to be under- or over-represented. The following chapter highlights the exceptional advances that have been achieved recently in splicing modulation with splice switching oligo

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Book 2018hroughput screening, pharmacological in vitro and in vivo investigations, drug-receptor interactions on the molecular level, structure-activity relationships, drug absorption, distribution, metabolism, elimination, toxicology and pharmacogenomics. In general, special volumes are edited by well known

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Approaches for the Discovery of Small Molecule Ligands Targeting microRNAs,ion, and dysregulation of miRNA expression has been implicated in many human disease states. Thus, therapeutically targeting miRNAs with small molecule ligands is of growing importance. Herein we focus on methods employed to discover small molecule miRNA ligands, their successes thus far, and future directions for the field.