ArticleNeuron2020
Antibody Therapy Targeting RAN Proteins Rescues C9 ALS/FTD Phenotypes in C9orf72 Mouse Model.
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Article in Neuron, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 69 papers, 1 of them a synthesis that pooled it.
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69citing papers in PubMed, 1 pooled it
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Who cites it
69 citing papers in PubMed, 1 synthesis or guideline pooled it, 109 citations in OpenAlex.
- Antibody-Based Therapeutic Interventions for Amyotrophic Lateral Sclerosis: A Systematic Literature Review.Frontiers in neuroscience · 2021Pooled it
- Two dipeptide repeat proteins are produced from mammalian telomeric RNA previously thought to be a long non-coding RNA.RNA biology · 2026Review
- Insights into X-linked Dystonia-Parkinsonism and Spinocerebellar Ataxia type 36 Through the Lens of Amyotrophic Lateral Sclerosis/Frontotemporal Dementia.Molecular neurobiology · 2026Review
- Designing polymer-peptide conjugates to target dipeptide repeat aggregates implicated in amyotrophic lateral sclerosis.Journal of materials chemistry. B · 2026Article
- Microglial Dysfunction Induced by C9ORF72 Dipeptide Repeat Proteins: Biomarker and Therapeutic Perspectives.International journal of molecular sciences · 2026Review
- Pharmacological rescue of mitochondrial dysfunction, neurite degeneration, and premature death of ALS and AD iPSC-derived neurons.bioRxiv : the preprint server for biology · 2026Article
- Autophagy-Neuroinflammation Axis in Neurodegenerative Diseases: Mechanisms and Therapeutic Potential.Cells · 2026Review
- Repeat-associated non-AUG translation as a common mechanism for the polyGln ataxias.Cell reports · 2026Article
- Article
- Cutting-edge treatments in amyotrophic lateral sclerosis: the role of molecular pathogenesis in targeted therapies.Stem cell research & therapy · 2025Review
- Inhibition of PolyGA Dipeptide Repeat Protein Aggregation by Nucleic Acid Aptamers in C9 Amyotrophic Lateral Sclerosis-Frontotemporal Dementia Models.Molecular neurobiology · 2025Article
- Blueprint of Collapse: Precision Biomarkers, Molecular Cascades, and the Engineered Decline of Fast-Progressing ALS.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
- Neuronal polyunsaturated fatty acids are protective in ALS/FTD.Nature neuroscience · 2025Article
- Review
- Amyotrophic lateral sclerosis caused by hexanucleotide repeat expansions in C9orf72: from genetics to therapeutics.The Lancet. Neurology · 2025Review
- Article
- A bacterial artificial chromosome mouse model of amyotrophic lateral sclerosis manifests 'space cadet syndrome' on two FVB backgrounds.Disease models & mechanisms · 2025Article
- CRISPR/Cas13d targeting suppresses repeat-associated non-AUG translation of C9orf72 hexanucleotide repeat RNA.The Journal of clinical investigation · 2024Article
- The prion-like effect and prion-like protein targeting strategy in amyotrophic lateral sclerosis.Heliyon · 2024Review
9 more citing papers are in PubMed but not listed here.
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Authors and funding
22 authors at 4 institutions in 2 countries.
Lien NguyenCenter for NeuroGenetics, Department of Molecular Genetics and Microbiology, Genetics Institute, McKnight Brain Institute, Norman Fixel Institute for Neurological Diseases, University of Florida, Gainesville, FL 32610, USA.
Fabio MontrasioNeurimmune AG, 8952 Schlieren, Switzerland.
Amrutha PattamattaCenter for NeuroGenetics, Department of Molecular Genetics and Microbiology, Genetics Institute, McKnight Brain Institute, Norman Fixel Institute for Neurological Diseases, University of Florida, Gainesville, FL 32610, USA.
Solaleh Khoramian TusiCenter for NeuroGenetics, Department of Molecular Genetics and Microbiology, Genetics Institute, McKnight Brain Institute, Norman Fixel Institute for Neurological Diseases, University of Florida, Gainesville, FL 32610, USA.
Olgert BardhiCenter for NeuroGenetics, Department of Molecular Genetics and Microbiology, Genetics Institute, McKnight Brain Institute, Norman Fixel Institute for Neurological Diseases, University of Florida, Gainesville, FL 32610, USA.
Kevin D MeyerNeurimmune AG, 8952 Schlieren, Switzerland; Institute for Regenerative Medicine-IREM, University of Zurich, 8952 Schlieren, Switzerland.
Lindsey HayesDepartment of Neurology, Johns Hopkins University, Baltimore, MD 21205, USA.
Katsuya NakamuraCenter for NeuroGenetics, Department of Molecular Genetics and Microbiology, Genetics Institute, McKnight Brain Institute, Norman Fixel Institute for Neurological Diseases, University of Florida, Gainesville, FL 32610, USA.
Monica Banez-CoronelCenter for NeuroGenetics, Department of Molecular Genetics and Microbiology, Genetics Institute, McKnight Brain Institute, Norman Fixel Institute for Neurological Diseases, University of Florida, Gainesville, FL 32610, USA.
Alyssa CoyneDepartment of Neurology, Johns Hopkins University, Baltimore, MD 21205, USA.
Shu GuoCenter for NeuroGenetics, Department of Molecular Genetics and Microbiology, Genetics Institute, McKnight Brain Institute, Norman Fixel Institute for Neurological Diseases, University of Florida, Gainesville, FL 32610, USA.
Lauren A LaboissonniereCenter for NeuroGenetics, Department of Molecular Genetics and Microbiology, Genetics Institute, McKnight Brain Institute, Norman Fixel Institute for Neurological Diseases, University of Florida, Gainesville, FL 32610, USA.
Yuanzheng GuNeuromuscular and Movement Disorders, Biogen, Cambridge, MA 02142, USA.
Saravanakumar NarayananNeuromuscular and Movement Disorders, Biogen, Cambridge, MA 02142, USA.
Benjamin SmithNeuromuscular and Movement Disorders, Biogen, Cambridge, MA 02142, USA.
Roger M NitschNeurimmune AG, 8952 Schlieren, Switzerland; Institute for Regenerative Medicine-IREM, University of Zurich, 8952 Schlieren, Switzerland.
Mark W KankelNeuromuscular and Movement Disorders, Biogen, Cambridge, MA 02142, USA.
Mia RusheNeuromuscular and Movement Disorders, Biogen, Cambridge, MA 02142, USA.
Jeffrey RothsteinDepartment of Neurology, Johns Hopkins University, Baltimore, MD 21205, USA.
Tao ZuCenter for NeuroGenetics, Department of Molecular Genetics and Microbiology, Genetics Institute, McKnight Brain Institute, Norman Fixel Institute for Neurological Diseases, University of Florida, Gainesville, FL 32610, USA.
Jan GrimmNeurimmune AG, 8952 Schlieren, Switzerland.
Laura P W RanumCenter for NeuroGenetics, Department of Molecular Genetics and Microbiology, Genetics Institute, McKnight Brain Institute, Norman Fixel Institute for Neurological Diseases, University of Florida, Gainesville, FL 32610, USA. Electronic address: ranum@ufl.edu.
University of Florida · USBiogen (United States) · USLight Chain Bioscience (Switzerland) · CHJohns Hopkins University · US
Funding
Molecular effects of metformin, PKR and TBI on C9orf72 ALS/FTDR01NS098819 · NINDS · UNIVERSITY OF FLORIDA · PI Laura P.W Ranum, Tao Zu · 2016 to 2026
$5.2MMolecular Genetic Characterization of SCA8R37NS040389 · NINDS · UNIVERSITY OF FLORIDA · PI RANUM, LAURA P.W · 2015 to 2021
$4.5MRole of Beta-III Spectrin in Spinocerebellar Ataxia Type 5R01NS056158 · NINDS · UNIVERSITY OF MINNESOTA · PI RANUM, LAURA P.W · 2007 to 2011
$2.8MMolecular effects of metformin, PKR and TBI on C9orf72 ALS/FTDRF1NS098819 · NINDS · UNIVERSITY OF FLORIDA · PI RANUM, LAURA P.W, ZU, TAO · 2022 to 2022
$2.2MFG-nucleoporins and nuclear transport disruption in C9ORF72-ALS/FTDK08NS104273 · NINDS · JOHNS HOPKINS UNIVERSITY · PI HAYES, LINDSEY RENAE · 2017 to 2021
$999kNINDS NIH HHS K08 NS104273NINDS NIH HHS R01 NS056158NINDS NIH HHS R01 NS098819NINDS NIH HHS R37 NS040389NINDS NIH HHS RF1 NS098819
Abstract
The intronic C9orf72 G4C2 expansion, the most common genetic cause of ALS and FTD, produces sense- and antisense-expansion RNAs and six dipeptide repeat-associated, non-ATG (RAN) proteins, but their roles in disease are unclear. We generated high-affinity human antibodies targeting GA or GP RAN proteins. These antibodies cross the blood-brain barrier and co-localize with intracellular RAN aggregates in C9-ALS/FTD BAC mice. In cells, α-GA
Indexed as
AgedAmyotrophic Lateral SclerosisAnimalsAntibodies, MonoclonalBrainC9orf72 ProteinCell Line, TumorDisease Models, AnimalFemaleFrontotemporal DementiaGene TargetingGenetic TherapyHEK293 CellsHumansMiceMice, Inbred C57BLAntibodies, MonoclonalC9orf72 ProteinC9orf72 protein, mouseran GTP-Binding ProteinRan protein, mouseRecombinant ProteinsBAC transgenic miceC9orf72 ALS/FTDG4C2 repeat expansionhuman antibodyimmunotherapymicrosatellite expansionproteasome and autophagyprotein co-aggregationRAN proteinsrepeat associated non-ATG (RAN) translation
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