ArticleCell2021
Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic.
Erratum issuedOpen access · bronzeAbstract read
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Article in Cell, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 149 papers.
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149citing papers in PubMed
17.4field-weighted citation impact, top 1% of its field
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Who cites it
149 citing papers in PubMed, 215 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
- Progranulin AAV gene therapy for frontotemporal dementia: translational studies and phase 1/2 trial interim results.Nature medicine · 2024Trial
- Batten disease protein CLN8 enables a non-canonical phospholipid synthesis pathway.Nature cell biology · 2026Article
- The Batten disease protein CLN8 is a stereospecific acyltransferase in bis(monoacylglycero)phosphate biosynthesis.Nature cell biology · 2026Article
- Lysosomal dysfunction in neurodegenerative disease.Nature reviews. Neurology · 2026Review
- Cellular and signalling mechanisms that regulate the blood-brain barrier.Nature reviews. Molecular cell biology · 2026Review
- Article
- Genetic frontotemporal degeneration across the lifespan? A critical appraisal of the neurodevelopmental hypothesis.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2026Review
- Hippocampal neuronal progranulin mediates estrogen‑deficiency‑induced affective vulnerability and lysosomal-autophagic dysfunction.Molecular psychiatry · 2026Article
- Progranulin deficiency perturbs lipid metabolism in white matter microglia.bioRxiv : the preprint server for biology · 2026Article
- Shared lipidome and proteome signatures of frontotemporal lobar degeneration and Alzheimer's disease.Research square · 2026Article
- CD98hc-targeted antibody shuttles for central nervous system delivery with broad cross-species reactivity.Nature biomedical engineering · 2026Article
- Lipid Metabolism, Lipogenesis, and Resistance to Third Generation EGFR-TKIs.Journal of clinical laboratory analysis · 2026Review
- Robust analytical methods for bis(monoacylglycero)phosphate profiling in health and disease.Nature protocols · 2026Review
- Microglial galectin-3 disrupts parvalbumin interneurons and hippocampal synchrony, driving cognitive deficits.Journal of neuroinflammation · 2026Article
- Cortical free-water imaging in familial frontotemporal dementia associated with MAPT, GRN, and C9orf72 pathogenic variants.Research square · 2026Article
- Progranulin deficiency in the brain activates an insulin signaling pathway that may promote neurodegeneration.iScience · 2026Article
- Targeting the PGRN-BMP Lysosomal Axis With NPs@PGRN Reverses Immunometabolic Dysfunction in Chronic Septic Arthritis.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Dissecting microglial contributions to neurodegenerative disease pathophysiology using human pluripotent stem cells.Stem cell reports · 2026Review
- Mucin-binding protein shuttles enable delivery of brain-targeted therapeutics.bioRxiv : the preprint server for biology · 2026Article
89 more citing papers are in PubMed but not listed here.
Corrections and comments
- Erratum issued
Authors and funding
53 authors at 3 institutions in 1 country.
Todd LoganDenali Therapeutics, South San Francisco, CA, USA.
Matthew J SimonDenali Therapeutics, South San Francisco, CA, USA.
Anil RanaDenali Therapeutics, South San Francisco, CA, USA.
Gerald M CherfDenali Therapeutics, South San Francisco, CA, USA.
Ankita SrivastavaDenali Therapeutics, South San Francisco, CA, USA.
Sonnet S DavisDenali Therapeutics, South San Francisco, CA, USA.
Ray Lieh Yoon LowDenali Therapeutics, South San Francisco, CA, USA.
Chi-Lu ChiuDenali Therapeutics, South San Francisco, CA, USA.
Meng FangDenali Therapeutics, South San Francisco, CA, USA.
Fen HuangDenali Therapeutics, South San Francisco, CA, USA.
Akhil BhallaDenali Therapeutics, South San Francisco, CA, USA.
Ceyda LlapashticaDenali Therapeutics, South San Francisco, CA, USA.
Rachel ProrokDenali Therapeutics, South San Francisco, CA, USA.
Michelle E PizzoDenali Therapeutics, South San Francisco, CA, USA.
Meredith E K CalvertDenali Therapeutics, South San Francisco, CA, USA.
Elizabeth W SunDenali Therapeutics, South San Francisco, CA, USA.
Jennifer Hsiao-NakamotoDenali Therapeutics, South San Francisco, CA, USA.
Yashas RajendraDenali Therapeutics, South San Francisco, CA, USA.
Katrina W LexaDenali Therapeutics, South San Francisco, CA, USA.
Devendra B SrivastavaDenali Therapeutics, South San Francisco, CA, USA.
Bettina van LengerichDenali Therapeutics, South San Francisco, CA, USA.
Junhua WangDenali Therapeutics, South San Francisco, CA, USA.
Yaneth Robles-ColmenaresDenali Therapeutics, South San Francisco, CA, USA.
Do Jin KimDenali Therapeutics, South San Francisco, CA, USA.
Joseph DuqueDenali Therapeutics, South San Francisco, CA, USA.
Melina LenserDenali Therapeutics, South San Francisco, CA, USA.
Timothy K EarrDenali Therapeutics, South San Francisco, CA, USA.
Hoang NguyenDenali Therapeutics, South San Francisco, CA, USA.
Roni ChauDenali Therapeutics, South San Francisco, CA, USA.
Buyankhishig TsogtbaatarDenali Therapeutics, South San Francisco, CA, USA.
Ritesh RaviDenali Therapeutics, South San Francisco, CA, USA.
Lukas L SkujaDenali Therapeutics, South San Francisco, CA, USA.
Hilda SolanoyDenali Therapeutics, South San Francisco, CA, USA.
Howard J RosenMemory and Aging Center, Department of Neurology, UCSF Weill Institute for Neurosciences, University of California, San Francisco, San Francisco, CA, USA; On behalf of the ALLFTD investigators.
Bradley F BoeveOn behalf of the ALLFTD investigators; Department of Neurology, College of Medicine, Mayo Clinic, Rochester, MN, USA.
Adam L BoxerMemory and Aging Center, Department of Neurology, UCSF Weill Institute for Neurosciences, University of California, San Francisco, San Francisco, CA, USA; On behalf of the ALLFTD investigators.
Hilary W HeuerMemory and Aging Center, Department of Neurology, UCSF Weill Institute for Neurosciences, University of California, San Francisco, San Francisco, CA, USA; On behalf of the ALLFTD investigators.
Mark S DennisDenali Therapeutics, South San Francisco, CA, USA.
Mihalis S KariolisDenali Therapeutics, South San Francisco, CA, USA.
Kathryn M MonroeDenali Therapeutics, South San Francisco, CA, USA.
Laralynne PrzybylaDenali Therapeutics, South San Francisco, CA, USA.
Pascal E SanchezDenali Therapeutics, South San Francisco, CA, USA.
Rene MeisnerDenali Therapeutics, South San Francisco, CA, USA.
Dolores DiazDenali Therapeutics, South San Francisco, CA, USA.
Kirk R HenneDenali Therapeutics, South San Francisco, CA, USA.
Ryan J WattsDenali Therapeutics, South San Francisco, CA, USA.
Anastasia G HenryDenali Therapeutics, South San Francisco, CA, USA.
Kannan GunasekaranDenali Therapeutics, South San Francisco, CA, USA.
Giuseppe AstaritaDenali Therapeutics, South San Francisco, CA, USA; Biochemistry and Molecular & Cellular Biology, Georgetown University, Washington, DC, USA.
Jung H SuhDenali Therapeutics, South San Francisco, CA, USA.
Joseph W LewcockDenali Therapeutics, South San Francisco, CA, USA.
Sarah L DeVosDenali Therapeutics, South San Francisco, CA, USA. Electronic address: devos@dnli.com.
Gilbert Di PaoloDenali Therapeutics, South San Francisco, CA, USA. Electronic address: dipaolo@dnli.com.
Denali Therapeutics (United States) · USUniversity of California, San Francisco · USMayo Clinic · US
Funding
Technology and Remote Assessment CoreU19AG063911 · NIA · MAYO CLINIC ROCHESTER · PI Bradley F Boeve · 2019 to 2026
$120.9MNational Centralized Repository for Alzheimer's Disease and Related Dementias (NCRAD)U24AG021886 · NIA · INDIANA UNIV-PURDUE UNIV AT INDIANAPOLIS · PI TATIANA M. FOROUD · 2002 to 2026
$119.8MTDP-43 Loss-of-Function: Biology to BiomarkersP01AG019724 · NIA · UNIVERSITY OF PENNSYLVANIA · PI Jennifer Merrilees · 2002 to 2026
$67.2MLongitudinal Evaluation of Familial Frontotemporal Dementia Subjects (LEFFTDS)U01AG045390 · NIA · MAYO CLINIC ROCHESTER · PI BOEVE, BRADLEY F, ROSEN, HOWARD J · 2014 to 2018
$16.9MTraining - The Frontotemporal Lobar Degeneration Clinical Research ConsortiumU54NS092089 · NINDS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI BOXER, ADAM L. · 2014 to 2018
$6.4MEye Movement Control in Normal Elderly and MCIR01AG031278 · NIA · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI BOXER, ADAM L. · 2009 to 2013
$1.2MNIA NIH HHS P01 AG019724NIA NIH HHS R01 AG031278NIA NIH HHS U01 AG045390NIA NIH HHS U19 AG063911NIA NIH HHS U24 AG021886NINDS NIH HHS U54 NS092089
Abstract
GRN mutations cause frontotemporal dementia (GRN-FTD) due to deficiency in progranulin (PGRN), a lysosomal and secreted protein with unclear function. Here, we found that Grn
Indexed as
AnimalsBiological ProductsBone Morphogenetic ProteinsBrainEndosomesFemaleFrontotemporal DementiaGliosisHumansInduced Pluripotent Stem CellsInflammationLipid MetabolismLipofuscinLysosomal Storage DiseasesLysosomesMacrophagesBiological ProductsBone Morphogenetic ProteinsGRN protein, humanGrn protein, mouseLipofuscinMembrane GlycoproteinsProgranulinsReceptors, ImmunologicReceptors, TransferrinTrem2 protein, mousegalectin-3GBALBPAlipidomicslipidslipofuscinlysobisphosphatidic acidlysosomemetabolomicsneurodegenerative disease
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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.