ArticleScience translational medicine2021
Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G
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Article in Science translational medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 43 papers.
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43citing papers in PubMed
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Who cites it
43 citing papers in PubMed.
- RIBOTAC-mediated degradation of hsa-microRNA-301a-3p suppresses TNBC bone metastasis.Science advances · 2026Article
- Engineered GM1 Intersects Between Mitochondrial and Synaptic Pathways to Ameliorate ALS Pathology.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Targeting RNA binding proteins with small-molecule inhibitors: Advances, challenges, and therapeutic opportunities.Acta pharmaceutica Sinica. B · 2026Review
- Induced proximity-based therapeutic modalities.Nature reviews. Drug discovery · 2026Review
- Deciphering the regulatory landscape of enhancer RNAs in health and disease.Signal transduction and targeted therapy · 2026Review
- RNA G-Quadruplex RIBOTAC-Mediated Targeted Degradation of lncRNA TERRA.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- A Live-Cell NanoBRET Assay to Monitor RNA-Protein Interactions and Their Inhibition by Small Molecules.ACS central science · 2025Article
- RNA-Focused DNA-Encoded Library Construction, Screening, and Integration of Docking Identify Bioactive Ligands of Pathogenic r(GbioRxiv : the preprint server for biology · 2025Article
- Targeted RNA Degradation as a Promising Therapeutic Strategy.International journal of molecular sciences · 2025Review
- Targeting neurodegenerative disease-associated protein aggregation with proximity-inducing modalities.Acta pharmacologica Sinica · 2025Review
- Review
- Design of an Orally Bioavailable Small Molecule That Modulates the Microtubule-Associated Protein Tau's Pre-mRNA Splicing.ACS chemical biology · 2025Article
- Therapeutic Approaches for C9ORF72-Related ALS: Current Strategies and Future Horizons.International journal of molecular sciences · 2025Review
- Mechanistic studies of small molecule ligands selective to RNA single G bulges.Nucleic acids research · 2025Article
- The Druggable Transcriptome Project: From Chemical Probes to Precision Medicines.Biochemistry · 2025Review
- High-throughput screen of 100 000 small molecules in C9ORF72 ALS neurons identifies spliceosome modulators that mobilize G4C2 repeat RNA into nuclear export and repeat associated non-canonical translation.Nucleic acids research · 2025Article
- Mapping small molecule-RNA binding sites via Chem-CLIP synergized with capillary electrophoresis and nanopore sequencing.Nucleic acids research · 2025Article
- Amyotrophic lateral sclerosis caused by hexanucleotide repeat expansions in C9orf72: from genetics to therapeutics.The Lancet. Neurology · 2025Review
- Exploring the therapeutic potential of modulating nonsense-mediated mRNA decay.RNA (New York, N.Y.) · 2025Review
- Kaempferol enhances ER-mitochondria coupling and protects motor neurons from mitochondrial dysfunction and ER stress in C9ORF72-ALS.Acta neuropathologica communications · 2025Article
Corrections and comments
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Authors and funding
24 authors.
Jessica A BushDepartment of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.ORCID 0000-0001-9474-1608
Haruo AikawaDepartment of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.ORCID 0000-0003-2536-900X
Rita FuerstDepartment of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
Yue LiDepartment of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
Andrei UrsuDepartment of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
Samantha M MeyerDepartment of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.ORCID 0000-0003-3353-6939
Raphael I BenhamouDepartment of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.ORCID 0000-0003-1743-0886
Jonathan L ChenDepartment of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.ORCID 0000-0001-7907-9732
Tanya KhanDepartment of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.ORCID 0000-0003-0323-8017
Sarah Wagner-GriffinDepartment of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.ORCID 0000-0003-4718-6909
Montina J Van MeterDepartment of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.ORCID 0000-0002-9313-2162
Yuquan TongDepartment of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.ORCID 0000-0003-0793-7747
Hailey OlafsonCenter for NeuroGenetics, College of Medicine, University of Florida, Gainesville, FL 32610, USA.
Kendra K McKeeCenter for NeuroGenetics, College of Medicine, University of Florida, Gainesville, FL 32610, USA.ORCID 0000-0002-1657-5998
Jessica L Childs-DisneyDepartment of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
Tania F GendronDepartment of Neuroscience, Mayo Clinic, 4500 San Pablo Road, Jacksonville, FL 32224, USA.ORCID 0000-0002-7335-2627
Yongjie ZhangDepartment of Neuroscience, Mayo Clinic, 4500 San Pablo Road, Jacksonville, FL 32224, USA.ORCID 0000-0002-9900-4313
Alyssa N CoyneRobert Packard Center for ALS Research, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA.ORCID 0000-0002-3658-5325
Eric T WangCenter for NeuroGenetics, College of Medicine, University of Florida, Gainesville, FL 32610, USA.ORCID 0000-0003-2655-5525
Ilyas YildirimDepartment of Chemistry and Biochemistry, Florida Atlantic University, Jupiter, FL 33458, USA.
Kye Won WangDepartment of Chemistry and Biochemistry, Florida Atlantic University, Jupiter, FL 33458, USA.ORCID 0000-0002-5718-0685
Leonard PetrucelliDepartment of Neuroscience, Mayo Clinic, 4500 San Pablo Road, Jacksonville, FL 32224, USA.ORCID 0000-0003-2959-129X
Jeffrey D RothsteinRobert Packard Center for ALS Research, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA.ORCID 0000-0003-2001-8470
Matthew D DisneyDepartment of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.ORCID 0000-0001-8486-1796
Funding
Expanding insights into FTD disease mechanismsR35NS097273 · NINDS · MAYO CLINIC JACKSONVILLE · PI PETRUCELLI, LEONARD · 2017 to 2024
$10.6MDesign of Precision Small Molecules Targeting RNA Repeating Transcripts to Manipulate and Study Disease BiologyR35NS116846 · NINDS · UNIVERSITY OF FLORIDA · PI Matthew D Disney · 2020 to 2026
$9.4MValidation of r(G4C2)exp-binding small molecules in vivo and c9FTD/ALS biomarker developmentP01NS099114 · NINDS · UNIVERSITY OF FLORIDA · PI DISNEY, MATTHEW D, PETRUCELLI, LEONARD · 2017 to 2021
$6.5MUsing a disease-affected cell to synthesize its own drugDP1NS096898 · NINDS · SCRIPPS FLORIDA · PI DISNEY, MATTHEW D · 2015 to 2019
$5.0MBruker Avance III HD 600 MHz NMR spectrometerS10OD021550 · OD · SCRIPPS FLORIDA · PI KOJETIN, DOUGLAS · 2017 to 2017
$600kNIH HHS S10 OD021550NINDS NIH HHS DP1 NS096898NINDS NIH HHS P01 NS099114NINDS NIH HHS R35 NS097273NINDS NIH HHS R35 NS116846
Abstract
The most common cause of amyotrophic lateral sclerosis and frontotemporal dementia (c9ALS/FTD) is an expanded G
Indexed as
Amyotrophic Lateral SclerosisFrontotemporal DementiaC9orf72 ProteinDNA Repeat ExpansionHumansRibonucleasesC9orf72 ProteinRibonucleases
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.