Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Topics Community

Article

Transcriptome-Wide Studies of RNA-Targeted Small Molecules Provide a Simple and Selective r(CUG)exp Degrader in Myotonic Dystrophy

26 Jun 2023

Abstract excerpt

High Resolution Image Download MS PowerPoint Slide Myotonic dystrophy type 1 (DM1) is caused by a highly structured RNA repeat expansion, r(CUG) exp, harbored in the 3′ untranslated region (3′ UTR) of dystrophia myotonica protein kinase ( DMPK ) mRNA and drives disease through a gain-of-function mechanism. A panel of low-molecular-weight fragments capable of reacting with RNA upon UV irradiation was studied for...

Source

Identifiers

PMID: 37521782
DOI: 10.1021/acscentsci.2c01223

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Community interpretation

Discuss the evidence

Ask a question, add context or challenge an interpretation. Community activity is separate from scientific evidence strength.

0

discussions

0

replies

Citations encouraged

Claims remain attributable.

No discussion yet

Be the first to add a careful, cited interpretation.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Transcriptome-Wide Studies of RNA-Targeted Small Molecules Provide a Simple and Selective r(CUG)exp Degrader in Myotonic DystrophyPMID 37521782Read ↗

Structure-Specific Cleavage of an RNA Repeat Expansion with a Dimeric Small Molecule Is Advantageous over Sequence-Specific Recognition by an Oligonucleotide.Semantic proximity Read ↗

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

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

A Druglike Small Molecule that Targets r(CCUG) Repeats in Myotonic Dystrophy Type 2 Facilitates Degradation by RNA Quality Control PathwaysSemantic proximity Read ↗

Furamidine Rescues Myotonic Dystrophy Type I Associated Mis-Splicing through Multiple MechanismsSemantic proximity Read ↗

Study of an RNA-Focused DNA-Encoded Library Informs Design of a Degrader of a r(CUG) Repeat ExpansionSemantic proximity Read ↗

Therapeutics development in myotonic dystrophy type 1Semantic proximity Read ↗

Robust CRISPR/dCas13 RNA blockers specifically perturb miRNA-target interactions and rescue type 1 myotonic dystrophy pathologySemantic proximity Read ↗

MBNL Sequestration by Toxic RNAs and RNA Misprocessing in the Myotonic Dystrophy BrainSemantic proximity Read ↗

Co-opting MBNL-dependent alternative splicing cassette exons to control gene therapy in myotonic dystrophySemantic proximity Read ↗

A chemoinformatics-guided platform for efficient discovery of RNA-binding small molecules: Proof-of-concept for myotonic dystrophy type 1Semantic proximity Read ↗

Mitigating RNA Toxicity in Myotonic Dystrophy using Small MoleculesSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.