ArticleProceedings of the National Academy of Sciences of the United States of America2018
Murine knockin model for progranulin-deficient frontotemporal dementia with nonsense-mediated mRNA decay.
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Article in Proceedings of the National Academy of Sciences of the United States of America, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 51 papers.
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51citing papers in PubMed
6.1field-weighted citation impact, top 3% of its field
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Who cites it
51 citing papers in PubMed, 72 citations in OpenAlex.
- Preclinical and clinical investigation of VES001, an oral small molecule sortilin receptor inhibitor for the treatment of frontotemporal dementia: a first-in-human randomized placebo-controlled phase 1 trial.Nature communications · 2026Trial
- Human TDP-43 expression worsens FTD-related phenotypes in progranulin-insufficient mice.Neurobiology of disease · 2026Article
- Transplantation of human iPSC-derived microglia ameliorates neuropathology and circuit dysfunction in progranulin-deficient mice.Molecular neurodegeneration · 2026Article
- Estimation of human brain fluid clearance using intrathecal imaging contrast agents.Fluids and barriers of the CNS · 2026Observational
- Transplantation of Human IPSC-derived Microglia Ameliorates Neuropathology and Circuit Dysfunction in Progranulin-Deficient Mice.Research square · 2026Article
- Transplantation of Human IPSC-derived Microglia Ameliorates Neuropathology and Circuit Dysfunction in Progranulin-Deficient Mice.bioRxiv : the preprint server for biology · 2026Article
- Monospecific antibodies reveal reduction of granulins in frontotemporal dementia caused byMolecular neurodegeneration advances · 2026Article
- The first report of ceroid lipofuscinosis type 11 in China: a novel mutation of GRN and updated clinical review.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025Review
- Delivering Progranulin to Astrocytic Lysosomes Promotes Growth of Co-Cultured Neurons.Journal of neurochemistry · 2025Article
- Targeting Granulin Haploinsufficiency in Frontotemporal Dementia: From Genetic Mechanisms to Therapeutics.International journal of molecular sciences · 2025Review
- The role of endolysosomal progranulin and TMEM106B in neurodegenerative diseases.Molecular neurodegeneration · 2025Review
- TFEB overexpression alleviates autophagy-lysosomal deficits caused by progranulin insufficiency.Scientific reports · 2025Article
- Amyotrophic lateral sclerosis and frontotemporal dementia mutation reduces endothelial TDP-43 and causes blood-brain barrier defects.Science advances · 2025Article
- Progranulin deficiency in the brain: the interplay between neuronal and non-neuronal cells.Translational neurodegeneration · 2025Review
- ASO-mediated knock-down of GPNMB in mutant-GRN and in Grn-deficient peripheral myeloid cells disrupts lysosomal function and immune responses.Molecular neurodegeneration · 2025Article
- Neuronal Ceroid Lipofuscinosis-Concepts, Classification, and Avenues for Therapy.CNS neuroscience & therapeutics · 2025Review
- Biochemical, Biomarker, and Behavioral Characterization of the GrnMolecular neurobiology · 2024Article
- CNS-wide repopulation by hematopoietic-derived microglia-like cells corrects progranulin deficiency in mice.Nature communications · 2024Article
- PhagoStat a scalable and interpretable end to end framework for efficient quantification of cell phagocytosis in neurodegenerative disease studies.Scientific reports · 2024Article
- Biochemical, biomarker, and behavioral characterization of thebioRxiv : the preprint server for biology · 2024Article
Corrections and comments
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Authors and funding
13 authors at 7 institutions in 1 country.
Andrew D NguyenDepartment of Genetics and Complex Diseases, Harvard T. H. Chan School of Public Health, Boston, MA 02115.ORCID 0000-0002-9492-9472
Thi A NguyenDepartment of Genetics and Complex Diseases, Harvard T. H. Chan School of Public Health, Boston, MA 02115.
Jiasheng ZhangDepartment of Pathology, University of California, San Francisco, CA 94143.
Swathi DevireddyDepartment of Cell Biology, Yale University School of Medicine, New Haven, CT 06510.
Ping ZhouInstitute of Cardiovascular Disease, Gladstone Institutes, San Francisco, CA 94158.
Anna M KarydasMemory and Aging Center, Department of Neurology, University of California, San Francisco, CA 94158.
Xialian XuInstitute of Cardiovascular Disease, Gladstone Institutes, San Francisco, CA 94158.
Bruce L MillerMemory and Aging Center, Department of Neurology, University of California, San Francisco, CA 94158.
Frank RigoNeuroscience Drug Discovery, Ionis Pharmaceuticals, Carlsbad, CA 92010.
Shawn M FergusonDepartment of Cell Biology, Yale University School of Medicine, New Haven, CT 06510.
Eric J HuangDepartment of Pathology, University of California, San Francisco, CA 94143.ORCID 0000-0002-5381-3801
Tobias C WaltherDepartment of Genetics and Complex Diseases, Harvard T. H. Chan School of Public Health, Boston, MA 02115; twalther@hsph.harvard.edu robert@hsph.harvard.edu.
Robert V FareseDepartment of Genetics and Complex Diseases, Harvard T. H. Chan School of Public Health, Boston, MA 02115; twalther@hsph.harvard.edu robert@hsph.harvard.edu.
Broad Institute · USGladstone Institutes · USHarvard University · USSan Francisco VA Medical Center · USUniversity of California, San Francisco · USYale University · USIonis Pharmaceuticals (United States) · US
Funding
PrPSc SPECIFIC INTERACTION WITH NOVEL PrP-Fc FUSION PROTEINSP50AG023501 · NIA · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI MILLER, BRUCE L · 2004 to 2018
$27.1MYale Alzheimer Disease Research CenterP50AG047270 · NIA · YALE UNIVERSITY · PI STRITTMATTER, STEPHEN M · 2015 to 2019
$8.3MMolecular mechanisms that match lysosome function to cellular demandR01GM105718 · NIGMS · YALE UNIVERSITY · PI FERGUSON, SHAWN · 2013 to 2022
$2.9MEXTRAMULAR RESEARCH FACILITIES CONSTRUCTIONC06RR018928 · NCRR · J. DAVID GLADSTONE INSTITUTES · PI MAHLEY, ROBERT W. · 2003 to 2003
$2.1MMapping Progranulin's Bioactivity: Implications for Disease and Potential TherapiesR00AG047339 · NIA · SAINT LOUIS UNIVERSITY · PI NGUYEN, ANDREW D. · 2018 to 2020
$728kMapping Progranulin's Bioactivity: Implications for Disease and Potential TherapiK99AG047339 · NIA · HARVARD SCHOOL OF PUBLIC HEALTH · PI NGUYEN, ANDREW D. · 2014 to 2015
$257kMechanisms of Progranulin Deficiency in Neuroinflammation and NeurodegenerationI01BX002978 · VA · VETERANS AFFAIRS MED CTR SAN FRANCISCO · PI HUANG, ERIC J · 2017 to 2020
–BLRD VA I01 BX002978Howard Hughes Medical InstituteNCRR NIH HHS C06 RR018928NIA NIH HHS K99 AG047339NIA NIH HHS P50 AG023501NIA NIH HHS P50 AG047270NIA NIH HHS R00 AG047339NIGMS NIH HHS R01 GM105718
Abstract
Frontotemporal dementia (FTD) is the most common neurodegenerative disorder in individuals under age 60 and has no treatment or cure. Because many cases of FTD result from
Indexed as
MutationAnimalsDisease Models, AnimalFibroblastsFrontotemporal DementiaGene Knock-In TechniquesGranulinsHumansIntercellular Signaling Peptides and ProteinsLysosomesMice, Inbred C57BLNonsense Mediated mRNA DecayOligonucleotides, AntisenseProgranulinsRNA, MessengerGranulinsGRN protein, humanGrn protein, mouseIntercellular Signaling Peptides and ProteinsOligonucleotides, AntisenseProgranulinsRNA, Messengerfrontotemporal dementialysosomeneurodegenerationnonsense-mediated mRNA decayprogranulin
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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.