RNA-Targeted Therapeutics.
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TLDR
RNA-targeted therapies represent a platform for drug discovery involving chemically modified oligonucleotides, a wide range of cellular RNAs, and a novel target-binding motif, Watson-Crick base pairing, which is efficient and supports approaching "undruggable" targets.About:
This article is published in Cell Metabolism.The article was published on 2018-04-03 and is currently open access. It has received 493 citations till now.read more
Citations
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Journal ArticleDOI
Therapeutic siRNA: state of the art.
TL;DR: In this review, the evolution of siRNA chemical modifications and their biomedical performance are comprehensively reviewed and all clinically explored and commercialized siRNA delivery platforms, including the GalNAc–siRNA conjugate, and their fundamental design principles are thoroughly discussed.
Journal ArticleDOI
Principles for targeting RNA with drug-like small molecules
TL;DR: In this article, the authors discuss principles for discovering small-molecule drugs that target RNA and argue that the overarching challenge is to identify appropriate target structures - namely, in disease-causing RNAs that have high information content and, consequently, appropriate ligand-binding pockets.
Journal Article
Eteplirsen for the Treatment of Duchenne Muscular Dystrophy (DMD) (S42.001)
Jay S. Charleston,F.J. Schnell,Johannes Dworzak,Cas Donoghue,J Lynch,Sarah Lewis,Lei Chen,Louise R. Rodino-Klapac,Zarife Sahenk,Jon Voss,U. DeAlwis,D. Frank,H Eliopoulos,J. Mendell +13 more
TL;DR: The present study used a double‐blind placebo‐controlled protocol to test eteplirsen's ability to induce dystrophin production and improve distance walked on the 6‐minute walk test.
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Epigenetics and epigenomics in diabetic kidney disease and metabolic memory
Mitsuo Kato,Rama Natarajan +1 more
TL;DR: The emerging role of epigenetics and epigenomics in DKD and the translational potential of candidate epigenetic factors and non-coding RNAs as biomarkers and drug targets for DKD are highlighted.
Journal ArticleDOI
IL-17 receptor–based signaling and implications for disease
TL;DR: New developments in IL-17 signaling and their pathophysiological implications are summarized, noting their normal protective roles directed against fungi and bacteria as well as against pathological conditions in inflammation and cancer.
References
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Journal ArticleDOI
Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
Andrew Fire,SiQun Xu,Mary K. Montgomery,Steven A. Kostas,Steven A. Kostas,Samuel E. Driver,Craig C. Mello +6 more
TL;DR: To their surprise, it was found that double-stranded RNA was substantially more effective at producing interference than was either strand individually, arguing against stochiometric interference with endogenous mRNA and suggesting that there could be a catalytic or amplification component in the interference process.
Journal ArticleDOI
Identification and characterization of a spinal muscular atrophy-determining gene
Suzie Lefebvre,Lydie Burglen,Sophie Reboullet,Olivier Clermont,Philippe Burlet,Louis Viollet,Bernard Bénichou,Corinne Cruaud,Philippe Millasseau,Massimo Zeviani,Denis Le Paslier,Jean Frézal,Daniel Cohen,Jean Weissenbach,Arnold Munnich,Judith Melki +15 more
TL;DR: The inverted duplication of a 500 kb element in normal chromosomes is described and the critical region is narrowed to 140 kb within the telomeric region, suggesting that this gene, termed the survival motor neuron (SMN) gene, is an SMA-determining gene.
Journal ArticleDOI
A species of small antisense RNA in posttranscriptional gene silencing in plants.
TL;DR: The 25-nucleotide antisense RNA detected in transgene-induced PTGS is likely synthesized from an RNA template and may represent the specificity determinant of PTGS.
Journal ArticleDOI
Argonaute2 Is the Catalytic Engine of Mammalian RNAi
Jidong Liu,Michelle A. Carmell,Michelle A. Carmell,Fabiola V. Rivas,Carolyn G. Marsden,J. Michael Thomson,Ji-Joon Song,Scott M. Hammond,Leemor Joshua-Tor,Gregory J. Hannon +9 more
TL;DR: The evidence supports a model in which Argonaute contributes “Slicer” activity to RISC, providing the catalytic engine for RNAi.
Journal ArticleDOI
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
Jürgen Soutschek,Akin Akinc,Birgit Bramlage,Klaus Charisse,Rainer Constien,Mary Donoghue,Sayda Elbashir,Anke Geick,Philipp Hadwiger,Jens Harborth,Matthias John,Venkitasamy Kesavan,Gary Lavine,Rajendra K. Pandey,Timothy Racie,Kallanthottathil G. Rajeev,Ingo Röhl,Ivanka Toudjarska,Gang Wang,Silvio Wuschko,David Bumcrot,Victor Koteliansky,Stefan Limmer,Muthiah Manoharan,Hans-Peter Vornlocher +24 more
TL;DR: In this article, chemically modified short interfering RNAs (siRNAs) were used to silence an endogenous gene encoding apolipoprotein B (apoB) after intravenous injection in mice.
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