ArticleNature medicine2022
Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide.
Open access · bronzeAbstract read
In one paragraph
Article in Nature medicine, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 121 papers, 1 of them a synthesis that pooled it.
0numbers the graph read from it
0cells of the map it votes in
121citing papers in PubMed, 1 pooled it
15.7field-weighted citation impact, top 1% of its field
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
121 citing papers in PubMed, 1 synthesis or guideline pooled it, 167 citations in OpenAlex.
- Pre-symptomatic radiological changes in frontotemporal dementia: propagation characteristics, predictive value and implications for clinical trials.Brain imaging and behavior · 2022Pooled it
- Insights into X-linked Dystonia-Parkinsonism and Spinocerebellar Ataxia type 36 Through the Lens of Amyotrophic Lateral Sclerosis/Frontotemporal Dementia.Molecular neurobiology · 2026Review
- Article
- SafeSense: An open-access safety atlas for antisense oligonucleotides adverse events in human.Molecular therapy. Nucleic acids · 2026Article
- Neural Stem Cell-Derived Extracellular Vesicles Improve CNS Delivery and Therapeutic Efficacy of Antisense Oligonucleotide in Amyotrophic Lateral Sclerosis.Journal of extracellular vesicles · 2026Article
- Toward the clinical application of long-read sequencing in repeat-expansion disorders.Nature genetics · 2026Review
- Antibody-oligonucleotide conjugates: an emerging modality for precision RNA therapeutics.Antibody therapeutics · 2026Review
- Intravenous administration of an engineered AAV9-gene-silencing vector suppresses human SOD1 and extends survival in an ALS mouse model.Nature communications · 2026Article
- Microglial Dysfunction Induced by C9ORF72 Dipeptide Repeat Proteins: Biomarker and Therapeutic Perspectives.International journal of molecular sciences · 2026Review
- Paying for precision: funding approaches for N-of-1 trials of individualized gene targeted therapies.Orphanet journal of rare diseases · 2026Review
- RNA targeting therapy for a prenatally enriched potassium channel associated with severe childhood epilepsy and premature death.Nature communications · 2026Article
- Direct targeting of C9ORF72 repeat RNA with fluorinated antisense oligonucleotides.Nucleic acids research · 2026Article
- Driving with Motor Neuron Disease: Disease-Specific Considerations, Multi-Domain Assessments and Support Strategies.Brain sciences · 2026Review
- ASO therapy rescues NOTCH2NLC GGC repeat expansion-induced genomic damage, 3D chromatin structural abnormalities, and senescence.Nature communications · 2026Article
- Antisense oligonucleotide-mediated knockdown therapy in two infants with severe KCNT1 epileptic encephalopathy.Nature medicine · 2026Article
- Gene Targeted Therapies for Neurodegenerative Disorders: Strategies and Implications in ALS and SMA.Genes · 2026Review
- The Role of Academic Medical Centers in Personalized Experimental Therapeutic Development: Key Considerations.Neurology · 2026Review
- Model selection in preclinical nucleic acid therapeutics research.Communications biology · 2026Review
- Skeletal Muscle Biomarkers of Amyotrophic Lateral Sclerosis: A Large-Scale, Multi-Cohort Proteomic Study.Annals of neurology · 2026Article
- Measuring neurofilament light in human plasma and cerebrospinal fluid: a comparison of five analytical immunoassays.Clinical chemistry and laboratory medicine · 2026Article
61 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on by
Authors and funding
21 authors at 4 institutions in 1 country.
Hélène Tran *Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
Michael P Moazami *RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.
Huiya YangDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
Diane McKenna-YasekDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
Catherine L DouthwrightDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
Courtney PintoDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
Jake MettervilleDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
Minwook ShinRNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.ORCID http://orcid.org/0000-0003-0496-5873
Nitasha SanilResearch Pharmacy, University of Massachusetts Medical School, Worcester, MA, USA.
Craig DooleyResearch Pharmacy, University of Massachusetts Medical School, Worcester, MA, USA.
Ajit PuriDepartment of Radiology, University of Massachusetts Medical School, Worcester, MA, USA.
Alexandra WeissDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
Nicholas WightmanDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA, USA.
Heather Gray-EdwardsDepartment of Radiology, University of Massachusetts Medical School, Worcester, MA, USA.
Miklos MarosfoiDepartment of Radiology, University of Massachusetts Medical School, Worcester, MA, USA.
Robert M KingDepartment of Radiology, University of Massachusetts Medical School, Worcester, MA, USA.ORCID http://orcid.org/0000-0002-5144-9110
Thomas KenderdineDepartment of Chemistry, University of Connecticut, Storrs, CT, USA.ORCID http://orcid.org/0000-0002-0533-1023
Daniele FabrisDepartment of Chemistry, University of Connecticut, Storrs, CT, USA.
Robert BowserDepartments of Neurology and Neurobiology, Barrow Neurological Institute, Phoenix, AZ, USA.ORCID http://orcid.org/0000-0001-5404-7259
Jonathan K WattsRNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA. jonathan.watts@umassmed.edu.ORCID http://orcid.org/0000-0001-5706-1734
Robert H BrownDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA, USA. robert.brown@umassmed.edu.ORCID http://orcid.org/0000-0001-6062-1528
University of Massachusetts Chan Medical School · USUniversity of Connecticut · USBarrow Neurological Institute · USWorcester Polytechnic Institute · US
Funding
University of Massachusetts Center for Clinical Science and Translational SupplementUL1TR001453 · NCATS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI LUZURIAGA, KATHERINE F · 2015 to 2024
$39.0MNext-generation antisense therapeutics for ALS and frontotemporal dementiaR01NS111990 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Robert H Brown, Jonathan K Watts · 2019 to 2026
$5.2MNCATS NIH HHS UL1 TR001453NINDS NIH HHS R01 NS111990
Abstract
Expansions of a G
Indexed as
MutationAnimalsC9orf72 ProteinFibroblastsHumansMiceMice, TransgenicNeuronsOligonucleotides, AntisenseC9orf72 ProteinOligonucleotides, Antisense
Identifiers
What OpenQuestion holds
Textmetadata
LicenceTDM
Read underepoch 390
Registered trials
None linked
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.