ArticleNature communications2022
CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro.
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In one paragraph
Article in Nature communications, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 53 papers.
0numbers the graph read from it
0cells of the map it votes in
53citing papers in PubMed
7.3field-weighted citation impact, top 2% of its field
What it found
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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.
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Who cites it
53 citing papers in PubMed, 66 citations in OpenAlex.
- 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
- Integrative ALS therapy: gene targeted therapy by base editing and potential exercise rehabilitation as synergistic strategies for functional recovery.Physical activity and nutrition · 2026Article
- Human iPSC‑based translational and reverse translational research for neurodegenerative diseases: emphasis on ALS and key advances.Japanese journal of radiology · 2026Review
- Dissecting the relationship between haplotypes around ATXN2 CAG repeats and the number of CAA interruptions by long-read sequencing.medRxiv : the preprint server for health sciences · 2026Article
- Exosome-like nanovesicles from acerola for CRISPR-Cas9 ribonucleoprotein delivery to the central nervous system.Molecular therapy. Nucleic acids · 2026Article
- Arrayed dual-gRNA CRISPR screening platform forMolecular therapy. Advances · 2026Article
- DNA and RNA editing for the therapy of human diseases: current status, challenges, and future prospects.Molecular biomedicine · 2026Review
- The Tale of the Guanosine Tract in Repeat Expansion Disorders.Molecular neurobiology · 2026Review
- Amyotrophic Lateral Sclerosis (ALS) Genetics and Microbiota: A Comprehensive Review.International journal of molecular sciences · 2026Review
- Engineered Cas9 exosome vesicles as a novel gene editing tool for targeted ASPN editing in osteoarthritis.Journal of nanobiotechnology · 2026Article
- Genetic therapies for neurological diseases.Pharmacological reviews · 2026Review
- Review
- 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
- Perspectives in Amyotrophic Lateral Sclerosis: Biomarkers, Omics, and Gene Therapy Informing Disease and Treatment.International journal of molecular sciences · 2025Review
- Targeting GeneInternational journal of molecular sciences · 2025Review
- Targets and Gene Therapy of ALS (Part 1).International journal of molecular sciences · 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
Corrections and comments
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Authors and funding
26 authors at 3 institutions in 1 country.
Katharina E MeijboomHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, 01605, USA.ORCID 0000-0003-0151-2983
Abbas AbdallahHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
Nicholas P FordhamHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, 01605, USA.ORCID 0000-0003-0059-2248
Hiroko NagaseHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
Tomás RodriguezRNA Therapeutics Institute and Program in Molecular Medicine, University of Massachusetts Medical School, Worcester, MA, 01605, USA.ORCID 0000-0002-8724-5427
Carolyn KrausRNA Therapeutics Institute and Program in Molecular Medicine, University of Massachusetts Medical School, Worcester, MA, 01605, USA.ORCID 0000-0001-7552-099X
Tania F GendronDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, 32224, USA.ORCID 0000-0002-7335-2627
Gopinath KrishnanDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
Rustam EsanovDepartment of Psychiatry & Behavioral Sciences, Center for Therapeutic Innovation, University of Miami Miller School of Medicine, Miami, FL, 33136, USA.
Nadja S AndradeDepartment of Psychiatry & Behavioral Sciences, Center for Therapeutic Innovation, University of Miami Miller School of Medicine, Miami, FL, 33136, USA.
Matthew J RybinDepartment of Psychiatry & Behavioral Sciences, Center for Therapeutic Innovation, University of Miami Miller School of Medicine, Miami, FL, 33136, USA.ORCID 0000-0001-8578-0891
Melina RamicDepartment of Psychiatry & Behavioral Sciences, Center for Therapeutic Innovation, University of Miami Miller School of Medicine, Miami, FL, 33136, USA.
Zachary D StephensDepartment of Quantitative Health Sciences. Mayo Clinic, Rochester, MN, 55905, USA.
Alireza EdrakiRNA Therapeutics Institute and Program in Molecular Medicine, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
Meghan T BlackwoodHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
Aydan KahrimanDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
Nils HenningerDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
Jean-Pierre A KocherDepartment of Quantitative Health Sciences. Mayo Clinic, Rochester, MN, 55905, USA.ORCID 0000-0002-0260-6773
Michael BenatarDepartment of Neurology, University of Miami Miller School of Medicine, Miami, FL, 33136, USA.
Michael H BrodskyDepartment of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA, 01605, USA.ORCID 0000-0001-7703-4872
Leonard PetrucelliDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, 32224, USA.
Fen-Biao GaoDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA, 01605, USA.ORCID 0000-0001-8873-5404
Erik J SontheimerRNA Therapeutics Institute and Program in Molecular Medicine, University of Massachusetts Medical School, Worcester, MA, 01605, USA.ORCID 0000-0002-0881-0310
Robert H BrownDepartment of Neurology, University of Massachusetts Medical School, Worcester, MA, 01605, USA.ORCID 0000-0001-6062-1528
Zane ZeierDepartment of Psychiatry & Behavioral Sciences, Center for Therapeutic Innovation, University of Miami Miller School of Medicine, Miami, FL, 33136, USA. zzeier@med.miami.edu.ORCID 0000-0003-0623-8610
Christian MuellerHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, 01605, USA. Christian.mueller4@sanofi.com.ORCID 0000-0003-3589-8749
University of Massachusetts Chan Medical School · USUniversity of Miami · USMayo Clinic in Florida · US
Funding
Expanding the chemical diversity of therapeutic oligonucleotides to treat neurodegenerative disordersR01NS104022 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI ANASTASIA KHVOROVA · 2017 to 2026
$6.0MNext-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
$5.2MInvestigating Pathogenic Mechanisms of Frontotemporal Dementia Caused by Mutations in CHMP2B and TBK1R37NS057553 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI GAO, FEN-BIAO · 2018 to 2024
$4.3MUnderstanding Frontotemporal Dementia Using Drosophila and iPSC ModelsR01NS101986 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Fen-Biao Gao · 2017 to 2026
$3.6MNINDS NIH HHS R01 NS101986NINDS NIH HHS R01 NS104022NINDS NIH HHS R01 NS111990NINDS NIH HHS R37 NS057553
Abstract
A GGGGCC
Indexed as
Amyotrophic Lateral SclerosisFrontotemporal DementiaAnimalsC9orf72 ProteinCRISPR-Cas SystemsDipeptidesDNA Repeat ExpansionMiceMotor NeuronsRNAC9orf72 ProteinDipeptidesRNA
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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.