ArticleCell2025
Molecular impact of antisense oligonucleotide therapy in C9orf72-associated ALS.
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Article in Cell, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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11citing papers in PubMed
–field-weighted citation impact
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Who cites it
11 citing papers in PubMed.
- Therapeutic frontiers in ALS: iPSC-based drug discovery, cell therapy, and gene therapy-Advances through 2026.Regenerative therapy · 2026Review
- Insights into X-linked Dystonia-Parkinsonism and Spinocerebellar Ataxia type 36 Through the Lens of Amyotrophic Lateral Sclerosis/Frontotemporal Dementia.Molecular neurobiology · 2026Review
- Regenerative strategies for ALS: stem cells and extracellular vesicles.Discover nano · 2026Review
- RAN Translation-Coupled Decay of theInternational journal of molecular sciences · 2026Article
- RAN translation as a dominant pathogenic axis in C9ORF72-associated ALS and FTD models.Molecular neurodegeneration · 2026Review
- Beyond neurofilaments: a multidimensional blood signature for amyotrophic lateral sclerosis.Brain communications · 2026Article
- Article
- NEDD8, stress granules, and amyotrophic lateral sclerosis: unveiling the therapeutic potential of the NEDP1 protease.Essays in biochemistry · 2025Review
- Chemically inducible antisense oligonucleotides for cell-specific gene silencing.RSC chemical biology · 2025Article
- Glioinflammation: disease-associated microglia and astrocytes in psychiatric disorders, neurodegeneration, and senescence.Frontiers in cellular neuroscience · 2025Review
- Emerging roles of primary cilia in the pathogenesis of amyotrophic lateral sclerosis.Frontiers in neuroscience · 2025Review
Corrections and comments
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Authors and funding
33 authors.
Zachary T McEachinDepartment of Human Genetics, Emory University, Atlanta, GA 30322, USA; Department of Cell Biology, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA. Electronic address: zmceach@emory.edu.
Mingee ChungDepartment of Human Genetics, Emory University, Atlanta, GA 30322, USA; Department of Cell Biology, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA.
Sabrina A StrattonDepartment of Human Genetics, Emory University, Atlanta, GA 30322, USA; Department of Cell Biology, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA.
Changhee HanDepartment of Human Genetics, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA.
Woo Jae KimDepartment of Human Genetics, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA.
Udit ShethMayo Clinic Graduate School of Biomedical Sciences, Mayo Clinic, Jacksonville, FL 32224, USA; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
Eleanor V ThomasGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA.
Ethan IssenbergDepartment of Human Genetics, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA.
Tanvi KamraDepartment of Human Genetics, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA.
Paola MerinoGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Pharmacology & Chemical Biology, Emory University, Atlanta, GA 30322, USA.
Yona LevitesGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Pharmacology & Chemical Biology, Emory University, Atlanta, GA 30322, USA.
Nisha RajDepartment of Human Genetics, Emory University, Atlanta, GA 30322, USA; Department of Cell Biology, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA.
Eric B DammerGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA.
Duc M DuongGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA.
Lingyan PingGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA.
Anantharaman ShantaramanGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA.
Adam N TrautwigGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA.
Joshna GadhaviGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA.
Ezana AssefaGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA.
Marla GearingGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Pathology, Emory University, Atlanta, GA 30322, USA.
Kaylor M KellyGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Pathology, Emory University, Atlanta, GA 30322, USA.
Shanu F RoemerDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
Michael DeTureDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
Seneshaw AsressGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA.
Thomas KukarGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Pharmacology & Chemical Biology, Emory University, Atlanta, GA 30322, USA.
Christina FournierDepartment of Neurology, Emory University, Atlanta, GA 30322, USA.
Dennis W DicksonMayo Clinic Graduate School of Biomedical Sciences, Mayo Clinic, Jacksonville, FL 32224, USA; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
Leonard PetrucelliMayo Clinic Graduate School of Biomedical Sciences, Mayo Clinic, Jacksonville, FL 32224, USA; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
Todd E GoldeGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Pharmacology & Chemical Biology, Emory University, Atlanta, GA 30322, USA.
Björn OskarssonDepartment of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.
Tania F GendronMayo Clinic Graduate School of Biomedical Sciences, Mayo Clinic, Jacksonville, FL 32224, USA; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
Nicholas T SeyfriedGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA. Electronic address: nseyfri@emory.edu.
Jonathan D GlassGoizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Pathology, Emory University, Atlanta, GA 30322, USA. Electronic address: jglas03@emory.edu.
Funding
The Goizueta Alzheimer's Disease Research CenterP30AG066511 · NIA · EMORY UNIVERSITY · PI Monica Willis Parker · 2020 to 2026
$29.0MProject 3: Neuropathology of the multi-proteinopathy of c9FTD/ALS.P01NS084974 · NINDS · MAYO CLINIC JACKSONVILLE · PI FRYER, JOHN DAVID · 2014 to 2024
$17.6MResolving the function of progranulin in lysosomal lipid metabolism and the etiology of Alzheimer's disease and frontotemporal dementiaRF1AG079318 · NIA · EMORY UNIVERSITY · PI KUKAR, THOMAS L · 2022 to 2025
$3.5MMolecular mechanisms of Progranulin in NeurodegenerationR01NS105971 · NINDS · EMORY UNIVERSITY · PI KUKAR, THOMAS L · 2018 to 2022
$1.9MMechanisms of chimeric DPR production and toxicity in c9ALS/FTDR01NS137434 · NINDS · EMORY UNIVERSITY · PI Zachary Thomas McEachin · 2024 to 2026
$1.9MNIA NIH HHS P30 AG066511NIA NIH HHS RF1 AG079318NINDS NIH HHS P01 NS084974NINDS NIH HHS R01 NS105971NINDS NIH HHS R01 NS137434
Abstract
C9orf72-associated amyotrophic lateral sclerosis (c9ALS) is caused by an intronic G
Indexed as
Amyotrophic Lateral SclerosisC9orf72 ProteinOligonucleotides, AntisenseAgedBiomarkersCentral Nervous SystemDNA-Binding ProteinsFemaleHumansMaleMiddle AgedSpinal CordBiomarkersC9orf72 ProteinC9orf72 protein, humanDNA-Binding ProteinsOligonucleotides, AntisenseTARDBP protein, humanamyotrophic lateral sclerosisantisense oligonucleotidesBIIB078C9orf72dipeptide repeat proteinsNULISAproteomicsTDP-43
Identifiers
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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 Socratic graph.