Progranulin as a therapeutic target in neurodegenerative diseases

Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic

(2021) Todd Logan et al.   CELL

Processing of progranulin into granulins involves multiple lysosomal proteases and is affected in frontotemporal lobar degeneration

(2021) Swetha Mohan et al.   Molecular Neurodegeneration

Microglial lysosome dysfunction contributes to white matter pathology and TDP-43 proteinopathy in GRN-associated FTD

(2021) Yanwei Wu et al.   Cell Reports

Plasma glial fibrillary acidic protein is raised in progranulin-associated frontotemporal dementia

(2020) Carolin Heller et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Reduced plasma progranulin levels are associated with the severity of Parkinson’s disease

(2020) Ming-Da Wang et al.   NEUROSCIENCE LETTERS

Premature termination codon readthrough upregulates progranulin expression and improves lysosomal function in preclinical models of GRN deficiency

(2020) Jonathan Frew et al.   Molecular Neurodegeneration

Altered serum protein levels in frontotemporal dementia and amyotrophic lateral sclerosis indicate calcium and immunity dysregulation

(2020) Jared S. Katzeff et al.   Scientific Reports

Progranulin and TMEM106B: when two become wan

(2020) Emma L Clayton et al.   EMBO REPORTS

Neurotoxic microglia promote TDP-43 proteinopathy in progranulin deficiency

(2020) Jiasheng Zhang et al.   NATURE

Network analysis of the progranulin-deficient mouse brain proteome reveals pathogenic mechanisms shared in human frontotemporal dementia caused by GRN mutations

(2020) Meixiang Huang et al.   Acta Neuropathologica Communications

Therapeutic landscape for Batten disease: current treatments and future prospects

(2019) Tyler B. Johnson et al.   Nature Reviews Neurology

Progranulin Stimulates the In Vitro Maturation of Pro-Cathepsin D at Acidic pH

(2019) Victoria J. Butler et al.   JOURNAL OF MOLECULAR BIOLOGY

Progranulin: A conductor of receptors orchestra, a chaperone of lysosomal enzymes and a therapeutic target for multiple diseases

(2019) Yazhou Cui et al.   CYTOKINE & GROWTH FACTOR REVIEWS

Neuronal ceroid lipofuscinosis type-11 in an adolescent

(2019) Mahesh Kamate et al.   BRAIN & DEVELOPMENT

Multi-Granulin Domain Peptides Bind to Pro-Cathepsin D and Stimulate Its Enzymatic Activity More Effectively Than Progranulin in Vitro

(2019) Victoria J. Butler et al.   BIOCHEMISTRY

Limbic-predominant age-related TDP-43 encephalopathy (LATE): consensus working group report

(2019) Peter T Nelson et al.   BRAIN

Disruption of RNA Metabolism in Neurological Diseases and Emerging Therapeutic Interventions

(2019) Julia K. Nussbacher et al.   NEURON

Enzyme replacement therapy with recombinant pro-CTSD (cathepsin D) corrects defective proteolysis and autophagy in neuronal ceroid lipofuscinosis

(2019) André R. A. Marques et al.   Autophagy

Age- and stress-associated C. elegans granulins impair lysosomal function and induce a compensatory HLH-30/TFEB transcriptional response

(2019) Victoria J. Butler et al.   PLoS Genetics

Age at symptom onset and death and disease duration in genetic frontotemporal dementia: an international retrospective cohort study

(2019) Katrina M Moore et al.   LANCET NEUROLOGY

Identification of novel risk loci, causal insights, and heritable risk for Parkinson's disease: a meta-analysis of genome-wide association studies

(2019) Mike A Nalls et al.   LANCET NEUROLOGY

Genetics of Alzheimer’s disease: where we are, and where we are going

(2019) Céline Bellenguez et al.   CURRENT OPINION IN NEUROBIOLOGY

Pathomechanisms of TDP-43 in neurodegeneration

(2018) Ju Gao et al.   JOURNAL OF NEUROCHEMISTRY

Progranulin Gene Therapy Improves Lysosomal Dysfunction and Microglial Pathology Associated with Frontotemporal Dementia and Neuronal Ceroid Lipofuscinosis

(2018) Andrew E. Arrant et al.   JOURNAL OF NEUROSCIENCE

Alzheimer’s Disease and Frontotemporal Dementia: The Current State of Genetics and Genetic Testing Since the Advent of Next-Generation Sequencing

(2018) Jill S. Goldman et al.   Molecular Diagnosis & Therapy

Microglia in neurodegeneration

(2018) Suzanne Hickman et al.   NATURE NEUROSCIENCE

Granulin A Synergizes with Cisplatin to Inhibit the Growth of Human Hepatocellular Carcinoma

(2018) Gan Qiao et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Progranulin reduces insoluble TDP-43 levels, slows down axonal degeneration and prolongs survival in mutant TDP-43 mice

(2018) Sander Beel et al.   Molecular Neurodegeneration

Progranulin-mediated deficiency of cathepsin D results in FTD and NCL-like phenotypes in neurons derived from FTD patients

(2017) Clarissa Valdez et al.   HUMAN MOLECULAR GENETICS

Progranulin deficiency causes impairment of autophagy and TDP-43 accumulation

(2017) Michael C. Chang et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Selective depletion of microglial progranulin in mice is not sufficient to cause neuronal ceroid lipofuscinosis or neuroinflammation

(2017) Terri L. Petkau et al.   Journal of Neuroinflammation

Neurotoxic reactive astrocytes are induced by activated microglia

(2017) Shane A. Liddelow et al.   NATURE

Sex differences in the prevalence of genetic mutations in FTD and ALS

(2017) Ashley F. Curtis et al.   NEUROLOGY

Cellular selectivity of AAV serotypes for gene delivery in neurons and astrocytes by neonatal intracerebroventricular injection

(2017) Sean L. Hammond et al.   PLoS One

Individuals with progranulin haploinsufficiency exhibit features of neuronal ceroid lipofuscinosis

(2017) Michael E. Ward et al.   Science Translational Medicine

Lipidomic and Transcriptomic Basis of Lysosomal Dysfunction in Progranulin Deficiency

(2017) Bret M. Evers et al.   Cell Reports

Differential Aging Analysis in Human Cerebral Cortex Identifies Variants in TMEM106B and GRN that Regulate Aging Phenotypes

(2017) Herve Rhinn et al.   Cell Systems

Progranulin Deficiency Promotes Circuit-Specific Synaptic Pruning by Microglia via Complement Activation

(2016) Hansen Lui et al.   CELL

Progranulin protects against exaggerated axonal injury and astrogliosis following traumatic brain injury

(2016) Lutz Menzel et al.   GLIA

The ubiquitin-proteasome system and autophagy: Coordinated and independent activities

(2016) Victoria Cohen-Kaplan et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

EphA2 is a functional receptor for the growth factor progranulin

(2016) Thomas Neill et al.   JOURNAL OF CELL BIOLOGY

Core neuropathological abnormalities in progranulin-deficient mice are penetrant on multiple genetic backgrounds

(2016) T.L. Petkau et al.   NEUROSCIENCE

Prosaposin is a regulator of progranulin levels and oligomerization

(2016) Alexandra M. Nicholson et al.   Nature Communications

Association Between Progranulin and Gaucher Disease

(2016) Jinlong Jian et al.   EBioMedicine

Prosaposin facilitates sortilin-independent lysosomal trafficking of progranulin

(2015) Xiaolai Zhou et al.   JOURNAL OF CELL BIOLOGY

The Progranulin Cleavage Products, Granulins, Exacerbate TDP-43 Toxicity and Increase TDP-43 Levels

(2015) D. A. Salazar et al.   JOURNAL OF NEUROSCIENCE

Haploinsufficiency of cathepsin D leads to lysosomal dysfunction and promotes cell-to-cell transmission of α-synuclein aggregates

(2015) E-J Bae et al.   Cell Death & Disease

Genetic modifiers in carriers of repeat expansions in the C9ORF72 gene

(2014) Marka van Blitterswijk et al.   Molecular Neurodegeneration

Progranulin protects against amyloid β deposition and toxicity in Alzheimer's disease mouse models

(2014) S Sakura Minami et al.   NATURE MEDICINE

Genetic basis and phenotypic correlations of the neuronal ceroid lipofusinoses

(2013) Varun Warrier et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Targeted manipulation of the sortilin–progranulin axis rescues progranulin haploinsufficiency

(2013) Wing C. Lee et al.   HUMAN MOLECULAR GENETICS

The neurotrophic properties of progranulin depend on the granulin E domain but do not require sortilin binding

(2013) Louis De Muynck et al.   NEUROBIOLOGY OF AGING

Promoter DNA methylation regulates progranulin expression and is altered in FTLD

(2013) Julia Banzhaf-Strathmann et al.   Acta Neuropathologica Communications

Strikingly Different Clinicopathological Phenotypes Determined by Progranulin-Mutation Dosage

(2012) Katherine R. Smith et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Progranulin: An emerging target for FTLD therapies

(2012) Jennifer Gass et al.   BRAIN RESEARCH

p62/sequestosome 1 binds to TDP-43 in brains with frontotemporal lobar degeneration with TDP-43 inclusions

(2012) Kunikazu Tanji et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Progranulin regulates neuronal outgrowth independent of Sortilin

(2012) Jennifer Gass et al.   Molecular Neurodegeneration

Exacerbated inflammatory responses related to activated microglia after traumatic brain injury in progranulin-deficient mice

(2012) Y. Tanaka et al.   NEUROSCIENCE

Hippocampal Sclerosis in the Elderly

(2011) Winnie C. Pao et al.   ALZHEIMER DISEASE & ASSOCIATED DISORDERS

Suberoylanilide Hydroxamic Acid (Vorinostat) Up-regulates Progranulin Transcription

(2011) Basar Cenik et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Low plasma progranulin levels in children with autism

(2011) Laila Y AL-Ayadhi et al.   Journal of Neuroinflammation

Core features of frontotemporal dementia recapitulated in progranulin knockout mice

(2011) N. Ghoshal et al.   NEUROBIOLOGY OF DISEASE

Progranulin, a Glycoprotein Deficient in Frontotemporal Dementia, Is a Novel Substrate of Several Protein Disulfide Isomerase Family Proteins

(2011) Sandra Almeida et al.   PLoS One

Genome-wide Screen Identifies rs646776 near Sortilin as a Regulator of Progranulin Levels in Human Plasma

(2010) Minerva M. Carrasquillo et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Progranulin expression in the developing and adult murine brain

(2010) Terri L. Petkau et al.   JOURNAL OF COMPARATIVE NEUROLOGY

Neuronal LRP1 Knockout in Adult Mice Leads to Impaired Brain Lipid Metabolism and Progressive, Age-Dependent Synapse Loss and Neurodegeneration

(2010) Q. Liu et al.   JOURNAL OF NEUROSCIENCE

Sortilin-Mediated Endocytosis Determines Levels of the Frontotemporal Dementia Protein, Progranulin

(2010) Fenghua Hu et al.   NEURON

Progranulin is Neurotrophic In Vivo and Protects against a Mutant TDP-43 Induced Axonopathy

(2010) Angela S. Laird et al.   PLoS One

The granulin gene family: from cancer to dementia

(2009) Andrew Bateman et al.   BIOESSAYS

Exaggerated inflammation, impaired host defense, and neuropathology in progranulin-deficient mice

(2009) Fangfang Yin et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Roles of Progranulin in Sexual Differentiation of the Developing Brain and Adult Neurogenesis

(2009) Masatoshi SUZUKI et al.   JOURNAL OF REPRODUCTION AND DEVELOPMENT

Cathepsin D expression level affects alpha-synuclein processing, aggregation, and toxicity in vivo

(2009) Valerie Cullen et al.   Molecular Brain

Progranulin functions as a neurotrophic factor to regulate neurite outgrowth and enhance neuronal survival

(2008) Philip Van Damme et al.   JOURNAL OF CELL BIOLOGY

Progranulin genetic variability contributes to amyotrophic lateral sclerosis

(2008) K. Sleegers et al.   NEUROLOGY