Matches in SemOpenAlex for { <https://semopenalex.org/work/W2325692787> ?p ?o ?g. }
Showing items 1 to 98 of
98
with 100 items per page.
- W2325692787 endingPage "2609" @default.
- W2325692787 startingPage "2605" @default.
- W2325692787 abstract "Potential RNA targets for chemical probes and therapeutic modalities are pervasive in the transcriptome. Oligonucleotide-based therapeutics are commonly used to target RNA sequence. Small molecules are emerging as a modality to target RNA structures selectively, but their development is still in its infancy. In this work, we compare the activity of oligonucleotides and several classes of small molecules that target the non-coding r(CCUG) repeat expansion (r(CCUG)exp) that causes myotonic dystrophy type 2 (DM2), an incurable disease that is the second-most common cause of adult onset muscular dystrophy. Small molecule types investigated include monomers, dimers, and multivalent compounds synthesized on-site by using RNA-templated click chemistry. Oligonucleotides investigated include phosphorothioates that cleave their target and vivo-morpholinos that modulate target RNA activity via binding. We show that compounds assembled on-site that recognize structure have the highest potencies amongst small molecules and are similar in potency to a vivo-morpholino modified oligonucleotide that targets sequence. These studies are likely to impact the design of therapeutic modalities targeting other repeats expansions that cause fragile X syndrome and amyotrophic lateral sclerosis, for example." @default.
- W2325692787 created "2016-06-24" @default.
- W2325692787 creator A5001563735 @default.
- W2325692787 creator A5017275370 @default.
- W2325692787 creator A5044229971 @default.
- W2325692787 date "2016-06-01" @default.
- W2325692787 modified "2023-10-14" @default.
- W2325692787 title "Comparison of small molecules and oligonucleotides that target a toxic, non-coding RNA" @default.
- W2325692787 cites W1513228388 @default.
- W2325692787 cites W1964997792 @default.
- W2325692787 cites W1971625140 @default.
- W2325692787 cites W1987435684 @default.
- W2325692787 cites W1994674028 @default.
- W2325692787 cites W2006167464 @default.
- W2325692787 cites W2008945738 @default.
- W2325692787 cites W2009372610 @default.
- W2325692787 cites W2025855695 @default.
- W2325692787 cites W2052021526 @default.
- W2325692787 cites W2056191842 @default.
- W2325692787 cites W2072383755 @default.
- W2325692787 cites W2083124133 @default.
- W2325692787 cites W2084091637 @default.
- W2325692787 cites W2109436341 @default.
- W2325692787 cites W2125968777 @default.
- W2325692787 cites W2130886644 @default.
- W2325692787 cites W2131447660 @default.
- W2325692787 cites W2136447541 @default.
- W2325692787 cites W2167126768 @default.
- W2325692787 cites W2319303211 @default.
- W2325692787 cites W2322093705 @default.
- W2325692787 cites W2949983130 @default.
- W2325692787 doi "https://doi.org/10.1016/j.bmcl.2016.04.025" @default.
- W2325692787 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/27117425" @default.
- W2325692787 hasPublicationYear "2016" @default.
- W2325692787 type Work @default.
- W2325692787 sameAs 2325692787 @default.
- W2325692787 citedByCount "14" @default.
- W2325692787 countsByYear W23256927872017 @default.
- W2325692787 countsByYear W23256927872018 @default.
- W2325692787 countsByYear W23256927872019 @default.
- W2325692787 countsByYear W23256927872020 @default.
- W2325692787 countsByYear W23256927872021 @default.
- W2325692787 countsByYear W23256927872022 @default.
- W2325692787 crossrefType "journal-article" @default.
- W2325692787 hasAuthorship W2325692787A5001563735 @default.
- W2325692787 hasAuthorship W2325692787A5017275370 @default.
- W2325692787 hasAuthorship W2325692787A5044229971 @default.
- W2325692787 hasConcept C104317684 @default.
- W2325692787 hasConcept C129312508 @default.
- W2325692787 hasConcept C136571751 @default.
- W2325692787 hasConcept C161624437 @default.
- W2325692787 hasConcept C185592680 @default.
- W2325692787 hasConcept C207001950 @default.
- W2325692787 hasConcept C2776878037 @default.
- W2325692787 hasConcept C54355233 @default.
- W2325692787 hasConcept C552990157 @default.
- W2325692787 hasConcept C55493867 @default.
- W2325692787 hasConcept C67705224 @default.
- W2325692787 hasConcept C70721500 @default.
- W2325692787 hasConcept C86803240 @default.
- W2325692787 hasConcept C95444343 @default.
- W2325692787 hasConceptScore W2325692787C104317684 @default.
- W2325692787 hasConceptScore W2325692787C129312508 @default.
- W2325692787 hasConceptScore W2325692787C136571751 @default.
- W2325692787 hasConceptScore W2325692787C161624437 @default.
- W2325692787 hasConceptScore W2325692787C185592680 @default.
- W2325692787 hasConceptScore W2325692787C207001950 @default.
- W2325692787 hasConceptScore W2325692787C2776878037 @default.
- W2325692787 hasConceptScore W2325692787C54355233 @default.
- W2325692787 hasConceptScore W2325692787C552990157 @default.
- W2325692787 hasConceptScore W2325692787C55493867 @default.
- W2325692787 hasConceptScore W2325692787C67705224 @default.
- W2325692787 hasConceptScore W2325692787C70721500 @default.
- W2325692787 hasConceptScore W2325692787C86803240 @default.
- W2325692787 hasConceptScore W2325692787C95444343 @default.
- W2325692787 hasFunder F4320308306 @default.
- W2325692787 hasIssue "11" @default.
- W2325692787 hasLocation W23256927871 @default.
- W2325692787 hasLocation W23256927872 @default.
- W2325692787 hasOpenAccess W2325692787 @default.
- W2325692787 hasPrimaryLocation W23256927871 @default.
- W2325692787 hasRelatedWork W1970173959 @default.
- W2325692787 hasRelatedWork W2021753748 @default.
- W2325692787 hasRelatedWork W2051801995 @default.
- W2325692787 hasRelatedWork W2078865641 @default.
- W2325692787 hasRelatedWork W2119463312 @default.
- W2325692787 hasRelatedWork W2141525074 @default.
- W2325692787 hasRelatedWork W2155344949 @default.
- W2325692787 hasRelatedWork W2174933528 @default.
- W2325692787 hasRelatedWork W2241766802 @default.
- W2325692787 hasRelatedWork W2325692787 @default.
- W2325692787 hasVolume "26" @default.
- W2325692787 isParatext "false" @default.
- W2325692787 isRetracted "false" @default.
- W2325692787 magId "2325692787" @default.
- W2325692787 workType "article" @default.