ArticleChemistry & biology2015
Engineering Duplex RNAs for Challenging Targets: Recognition of GGGGCC/CCCCGG Repeats at the ALS/FTD C9orf72 Locus.
Article in Chemistry & biology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed, 27 citations in OpenAlex.
- Direct targeting of C9ORF72 repeat RNA with fluorinated antisense oligonucleotides.Nucleic acids research · 2026Article
- Article
- Nuclear Argonaute:miRNA complexes recognize target sequences within chromatin-associated RNA and silence gene expression.Nucleic acids research · 2025Article
- Therapeutic targeting of RNA for neurological and neuromuscular disease.Genes & development · 2024Review
- Gene Therapy in Amyotrophic Lateral Sclerosis.Cells · 2022Review
- Gene therapy for ALS: A review.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- Analyzing pre-symptomatic tissue to gain insights into the molecular and mechanistic origins of late-onset degenerative trinucleotide repeat disease.Nucleic acids research · 2020Article
- Development of disease-modifying drugs for frontotemporal dementia spectrum disorders.Nature reviews. Neurology · 2020Review
- Structural Diversity of Sense and Antisense RNA Hexanucleotide Repeats Associated with ALS and FTLD.Molecules (Basel, Switzerland) · 2020Article
- Recent advances in understanding amyotrophic lateral sclerosis and emerging therapies.Faculty reviews · 2020Review
- A Peptidylic Inhibitor for NeutralizingMolecular therapy. Nucleic acids · 2019Article
- Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy.Molecular therapy. Nucleic acids · 2019Article
- Duplex RNAs and ss-siRNAs Block RNA Foci Associated with Fuchs' Endothelial Corneal Dystrophy.Nucleic acid therapeutics · 2019Article
- Artificial MicroRNAs Targeting C9orf72 Can Reduce Accumulation of Intra-nuclear Transcripts in ALS and FTD Patients.Molecular therapy. Nucleic acids · 2019Article
- Molecular Mechanisms of Neurodegeneration Related toBehavioural neurology · 2019Review
- Recognition of c9orf72 Mutant RNA by Single-Stranded Silencing RNAs.Nucleic acid therapeutics · 2017Article
- c9orf72 Disease-Related Foci Are Each Composed of One Mutant Expanded Repeat RNA.Cell chemical biology · 2017Article
Corrections and comments
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Authors and funding
5 authors at 2 institutions in 1 country.
Funding
Abstract
A GGGGCC expansion within an intronic region of the C9orf72 gene forms RNA foci that are associated with one-third of familial amyotrophic lateral sclerosis and one-quarter of frontotemporal dementia. The C9orf72 locus also expresses an antisense transcript with a CCCCGG expansion that forms foci and may contribute to disease. Synthetic agents that bind these hexanucleotide repeats and block foci would be leads for therapeutic discovery. We have engineered duplex RNAs to enable them to recognize difficult C/G targets. Recognition inhibits foci formed by both GGGGCC and CCCCGG RNA. Our findings show that a single duplex RNA can be used to recognize both disease-related C9orf72 transcripts. More broadly, we extend RNAi to previously inaccessible C/G sequences and provide another example of target recognition in human cells by nuclear RNAi.
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Registered trials
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