ArticleNature2020
Suppression of proteolipid protein rescues Pelizaeus-Merzbacher disease.
Open access · greenAbstract read
In one paragraph
Article in Nature, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 49 papers.
0numbers the graph read from it
0cells of the map it votes in
49citing papers in PubMed
3.4field-weighted citation impact, top 6% 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
49 citing papers in PubMed, 75 citations in OpenAlex.
- Beyond the Wiring: White Matter as a Dynamic Regulator of Brain Function and Disease.Neuroscience bulletin · 2026Review
- Beyond Neurodegeneration: White Matter Vacuolation as a Primary Myelin Defect.International journal of molecular sciences · 2026Review
- From insights to innovations: evaluating preclinical paradigms in demyelinating disease therapeutics.Lab animal · 2026Review
- Spatially concentrated adenine base editors efficiently correct PLP1 mutations in oligodendrocytes.Nucleic acids research · 2026Article
- Pelizaeus-Merzbacher disease in children: A case report.Radiology case reports · 2026Article
- Integrated stress response inhibition prolongs the lifespan of a Pelizaeus-Merzbacher disease mouse model by increasing oligodendrocyte survival.Nature communications · 2025Article
- Comparison ofMolecular therapy. Nucleic acids · 2025Article
- Article
- Stem cell and gene therapies for leukodystrophies.Molecular therapy. Methods & clinical development · 2025Review
- A copy number variant overlapping the 3'UTR of PLP1 causes spastic paraplegia.Journal of human genetics · 2025Article
- Transcriptomic Profiling of Zebrafish Mutant forInternational journal of molecular sciences · 2025Article
- Novel potential neuroprotective targets for DengZhanXiXin injection in middle cerebral artery occlusion rats recommended by quantitative proteomics and simulated docking.Frontiers in neuroscience · 2025Article
- PLP1-Targeting Antisense Oligonucleotides Improve FOXG1 Syndrome Mice.International journal of molecular sciences · 2024Article
- Gene therapy for the leukodystrophies: From preclinical animal studies to clinical trials.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024Review
- A mouse model of the protease-activated receptor 4 Pro310Leu variant has reduced platelet reactivity.Journal of thrombosis and haemostasis : JTH · 2024Article
- Homozygous EPRS1 missense variant causing hypomyelinating leukodystrophy-15 alters variant-distal mRNA mNature communications · 2024Article
- Pervasive environmental chemicals impair oligodendrocyte development.Nature neuroscience · 2024Article
- Pelizaeus-Merzbacher disease: on the cusp of myelin medicine.Trends in molecular medicine · 2024Review
- ASOs are an effective treatment for disease-associated oligodendrocyte signatures in premanifest and symptomatic SCA3 mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Recognizing Alzheimer's disease from perspective of oligodendrocytes: Phenomena or pathogenesis?CNS neuroscience & therapeutics · 2024Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
21 authors at 4 institutions in 1 country.
Matthew S ElittDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA.ORCID http://orcid.org/0000-0002-7296-164X
Lilianne BarbarDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA.ORCID http://orcid.org/0000-0001-9887-4248
H Elizabeth ShickDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA.
Berit E PowersIonis Pharmaceuticals, Carlsbad, CA, USA.
Yuka Maeno-HikichiDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA.
Mayur MadhavanDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA.
Kevin C AllanDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA.
Baraa S NawashDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA.
Artur S GevorgyanDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA.
Stevephen HungDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA.
Zachary S NevinDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA.ORCID http://orcid.org/0000-0003-4987-924X
Hannah E OlsenDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA.
Midori HitomiLerner Research Institute, Cleveland Clinic, Cleveland, OH, USA.
Daniela M SchlatzerCase Center for Proteomics and Bioinformatics, Department of Medicine, Case Western Reserve University School of Medicine, Cleveland, OH, USA.
Hien T ZhaoIonis Pharmaceuticals, Carlsbad, CA, USA.
Adam SwayzeIonis Pharmaceuticals, Carlsbad, CA, USA.
David F LePageDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA.
Weihong JiangDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA.
Ronald A ConlonDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA.
Frank RigoIonis Pharmaceuticals, Carlsbad, CA, USA.
Paul J TesarDepartment of Genetics and Genome Sciences, Case Western Reserve University School of Medicine, Cleveland, OH, USA. paul.tesar@case.edu.ORCID http://orcid.org/0000-0003-1532-3155
University School · USIonis Pharmaceuticals (United States) · USCase Western Reserve University · USCleveland Clinic Lerner College of Medicine · US
Funding
TUMOR METABOLISM PROGRAMP30CA043703 · NCI · CASE WESTERN RESERVE UNIVERSITY · PI MARK R CHANCE · 1987 to 2026
$142.3MMEDICAL SCIENTIST TRAINING PROGRAMT32GM007250 · NIGMS · CASE WESTERN RESERVE UNIVERSITY · PI HUANG, ALEX YEE-CHEN · 1985 to 2023
$33.4MModulating Glial Fate and Function in Development and DiseaseR35NS116842 · NINDS · CASE WESTERN RESERVE UNIVERSITY · PI Paul Joseph Tesar · 2020 to 2026
$5.3MNRSA Training CoreTL1TR002549 · NCATS · CASE WESTERN RESERVE UNIVERSITY · PI HARDING, CLIFFORD V · 2018 to 2022
$2.9MTraining in Neurodegenerative DiseasesT32NS077888 · NINDS · CASE WESTERN RESERVE UNIVERSITY · PI Xiongwei Zhu · 2013 to 2026
$1.8MHuman iPSC and glial chimeric modeling of Pelizaeus-Merzbacher DiseaseR01NS093357 · NINDS · CASE WESTERN RESERVE UNIVERSITY · PI TESAR, PAUL JOSEPH, WINDREM, MARTHA SHERRILL · 2015 to 2018
$1.5MOrbitrap Fusion Lumos Tribrid Mass SpectrometerS10OD026882 · OD · CASE WESTERN RESERVE UNIVERSITY · PI CHANCE, MARK R · 2019 to 2019
$1.0MAcquisition of a Leica TCS SP5 Confocal Microscope For A Multi-User FacilityS10OD016164 · OD · CASE WESTERN RESERVE UNIVERSITY · PI CONRAD, PATRICIA A · 2013 to 2013
$597kHamamatsu Nanozoomer S60 Digital Whole Slide ScannerS10OD024981 · OD · CASE WESTERN RESERVE UNIVERSITY · PI IMANISHI, YOSHIKAZU · 2018 to 2018
$303kIdentifying molecular and cellular deficits in a human model of myelin diseaseF30HD084167 · NICHD · CASE WESTERN RESERVE UNIVERSITY · PI NEVIN, ZACHARY SCOTT · 2015 to 2018
$163kMechanism of hypoxia mediated failure of oligodendrocyte generationF30HD096784 · NICHD · CASE WESTERN RESERVE UNIVERSITY · PI ALLAN, KEVIN CAMERON · 2019 to 2022
$163kNCATS NIH HHS TL1 TR002549NCI NIH HHS P30 CA043703NICHD NIH HHS F30 HD084167NICHD NIH HHS F30 HD096784NIGMS NIH HHS T32 GM007250NIH HHS S10 OD016164NIH HHS S10 OD024981NIH HHS S10 OD026882NINDS NIH HHS R01 NS093357NINDS NIH HHS R35 NS116842NINDS NIH HHS T32 NS077888
Abstract
Mutations in PLP1, the gene that encodes proteolipid protein (PLP), result in failure of myelination and neurological dysfunction in the X-chromosome-linked leukodystrophy Pelizaeus-Merzbacher disease (PMD)
Indexed as
Disease Models, AnimalAnimalsCRISPR-Cas SystemsFemaleGene EditingHypoxiaMaleMiceMice, Mutant StrainsMotor ActivityMyelin Proteolipid ProteinMyelin SheathOligodendrogliaOligonucleotides, AntisensePelizaeus-Merzbacher DiseasePoint MutationMyelin Proteolipid ProteinOligonucleotides, AntisensePlp1 protein, mouse
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.