ArticleNature communications2025
Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo.
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In one paragraph
Article in Nature communications, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
0numbers the graph read from it
0cells of the map it votes in
17citing papers in PubMed
–field-weighted citation impact
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
17 citing papers in PubMed.
- Beyond DNA editing: how Cas13 redefined programmable RNA manipulation and what still limits its therapeutic promise.Nucleic acids research · 2026Review
- DNA and RNA editing for the therapy of human diseases: current status, challenges, and future prospects.Molecular biomedicine · 2026Review
- Genetic epidemiology of C9orf72 repeat expansion associated amyotrophic lateral sclerosis in Hungary.Molecular medicine (Cambridge, Mass.) · 2026Article
- Review
- Gene Targeted Therapies for Neurodegenerative Disorders: Strategies and Implications in ALS and SMA.Genes · 2026Review
- Review
- Article
- Nanomedicine-enhanced delivery of CRISPR-Cas13 for RNA editing in C9orf72-associated ALS.Annals of medicine and surgery (2012) · 2025Article
- Review
- Targeting Granulin Haploinsufficiency in Frontotemporal Dementia: From Genetic Mechanisms to Therapeutics.International journal of molecular sciences · 2025Review
- CTAD taskforce: genetic therapies in Alzheimer's disease.The journal of prevention of Alzheimer's disease · 2025Review
- Blueprint of Collapse: Precision Biomarkers, Molecular Cascades, and the Engineered Decline of Fast-Progressing ALS.International journal of molecular sciences · 2025Review
- CRISPR in Neurodegenerative Diseases Treatment: An Alternative Approach to Current Therapies.Genes · 2025Review
- Therapeutic Approaches for C9ORF72-Related ALS: Current Strategies and Future Horizons.International journal of molecular sciences · 2025Review
- Induced Pluripotent (iPSC) and Mesenchymal (MSC) Stem Cells for In Vitro Disease Modeling and Regenerative Medicine.International journal of molecular sciences · 2025Review
- Amyotrophic lateral sclerosis caused by hexanucleotide repeat expansions in C9orf72: from genetics to therapeutics.The Lancet. Neurology · 2025Review
- The role of autophagy in the pathogenesis and treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).Autophagy reports · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
30 authors.
Liam Kempthorne *UK Dementia Research Institute at UCL, London, WC1E 6BT, UK.
Deniz Vaizoglu *UK Dementia Research Institute at UCL, London, WC1E 6BT, UK.
Alexander J Cammack *UK Dementia Research Institute at UCL, London, WC1E 6BT, UK.
Mireia CarcoléUK Dementia Research Institute at UCL, London, WC1E 6BT, UK.ORCID http://orcid.org/0000-0001-5054-9016
Martha J RobertsUK Dementia Research Institute at UCL, London, WC1E 6BT, UK.ORCID http://orcid.org/0009-0003-6441-6865
Alla MikheenkoUK Dementia Research Institute at UCL, London, WC1E 6BT, UK.
Alessia FisherUK Dementia Research Institute at UCL, London, WC1E 6BT, UK.
Pacharaporn SuklaiUK Dementia Research Institute at UCL, London, WC1E 6BT, UK.
Bhavana MuralidharanUK Dementia Research Institute at UCL, London, WC1E 6BT, UK.
François KrollDepartment of Cell and Developmental Biology, University College London, London, WC1E 6BT, UK.ORCID http://orcid.org/0000-0001-9908-2648
Thomas G MoensVIB-KU Center for Brain and Disease Research, Leuven, 3001, Belgium.
Lidia YshiiVIB-KU Center for Brain and Disease Research, Leuven, 3001, Belgium.
Stijn VerschorenVIB-KU Center for Brain and Disease Research, Leuven, 3001, Belgium.
Benedikt V HölblingUK Dementia Research Institute at UCL, London, WC1E 6BT, UK.
Francisco C MoreiraDepartment of Clinical & Experimental Epilepsy, UCL Queen Square Institute of Neurology, London, WC1N 3BG, UK.ORCID http://orcid.org/0000-0002-7148-4386
Eszter KatonaUK Dementia Research Institute at UCL, London, WC1E 6BT, UK.
Rachel ConeysUK Dementia Research Institute at UCL, London, WC1E 6BT, UK.
Paula de OliveiraUK Dementia Research Institute at UCL, London, WC1E 6BT, UK.
Yong-Jie ZhangDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, 32224, USA.
Karen JansenDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, 32224, USA.
Lillian M DaughrityDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, 32224, USA.ORCID http://orcid.org/0000-0003-4644-7265
Alexander McGownSheffield Institute for Translational Neuroscience, University of Sheffield, Sheffield, S10 2HQ, UK.
Tennore M RameshSheffield Institute for Translational Neuroscience, University of Sheffield, Sheffield, S10 2HQ, UK.
Ludo Van Den BoschVIB-KU Center for Brain and Disease Research, Leuven, 3001, Belgium.
Gabriele LignaniDepartment of Clinical & Experimental Epilepsy, UCL Queen Square Institute of Neurology, London, WC1N 3BG, UK.ORCID http://orcid.org/0000-0002-3963-9296
Ahad A RahimUCL School of Pharmacy, University College London, London, WC1N 1AX, UK.
Alyssa N CoyneDepartment of Neurology, Johns Hopkins University, Baltimore, USA.ORCID http://orcid.org/0000-0002-3658-5325
Leonard PetrucelliDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, 32224, USA.
Jason RihelDepartment of Cell and Developmental Biology, University College London, London, WC1E 6BT, UK.ORCID http://orcid.org/0000-0003-4067-2066
Adrian M IsaacsUK Dementia Research Institute at UCL, London, WC1E 6BT, UK. a.isaacs@ucl.ac.uk.ORCID http://orcid.org/0000-0002-6820-5534
Funding
EC | EU Framework Programme for Research and Innovation H2020 | H2020 Priority Excellent Science | H2020 European Research Council (H2020 Excellent Science - European Research Council) 648716 - C9NDWellcome TrustWellcome Trust (Wellcome) 217150/Z/19/Z
Abstract
The most common genetic cause of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) is an intronic G
Indexed as
Amyotrophic Lateral SclerosisC9orf72 ProteinCRISPR-Cas SystemsFrontotemporal DementiaInduced Pluripotent Stem CellsRNA, AntisenseAnimalsDisease Models, AnimalDNA Repeat ExpansionGenetic TherapyHEK293 CellsHumansMiceNeuronsC9orf72 ProteinC9orf72 protein, humanRNA, Antisense
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Read underepoch 390
Registered trials
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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.