[HTML][HTML] Progranulin as a therapeutic target in neurodegenerative diseases

H Rhinn, N Tatton, S McCaughey, M Kurnellas… - Trends in …, 2022 - cell.com
Progranulin (PGRN, encoded by the GRN gene) plays a key role in the development,
survival, function, and maintenance of neurons and microglia in the mammalian brain. It …

Increased caspase activation and decreased TDP‐43 solubility in progranulin knockout cortical cultures

G Kleinberger, H Wils, P Ponsaerts… - Journal of …, 2010 - Wiley Online Library
J. Neurochem.(2010) 115, 735–747. Abstract Null mutations in progranulin (GRN) are
associated with frontotemporal lobar degeneration characterized by intraneuronal …

Progranulin haploinsufficiency causes biphasic social dominance abnormalities in the tube test

AE Arrant, AJ Filiano, BA Warmus… - Genes, Brain and …, 2016 - Wiley Online Library
Loss‐of‐function mutations in progranulin (GRN) are a major autosomal dominant cause of
frontotemporal dementia (FTD), a neurodegenerative disorder in which social behavior is …

An 8‐week, open‐label, dose‐finding study of nimodipine for the treatment of progranulin insufficiency from GRN gene mutations

SJ Sha, ZA Miller, S Min, Y Zhou… - … Research & Clinical …, 2017 - Wiley Online Library
Introduction Frontotemporal lobar degeneration–causing mutations in the progranulin (GRN)
gene reduce progranulin protein (PGRN) levels, suggesting that restoring PGRN in mutation …

Progranulin promotes neurite outgrowth and neuronal differentiation by regulating GSK-3β

X Gao, AP Joselin, L Wang, A Kar, P Ray, A Bateman… - Protein & cell, 2010 - Springer
Progranulin (PGRN) has recently emerged as a key player in a subset of frontotemporal
dementias (FTD). Numerous mutations in the progranulin gene have been identified in …

[HTML][HTML] Progranulin AAV gene therapy for frontotemporal dementia: translational studies and phase 1/2 trial interim results

J Sevigny, O Uspenskaya, LD Heckman, LC Wong… - Nature Medicine, 2024 - nature.com
GRN mutations cause progranulin haploinsufficiency, which eventually leads to
frontotemporal dementia (FTD-GRN). PR006 is an investigational gene therapy delivering …

Progranulin reduction is associated with increased tau phosphorylation in P301L tau transgenic mice

M Hosokawa, T Arai… - … of Neuropathology & …, 2015 - academic.oup.com
Granulin (GRN) mutations have been identified in familial frontotemporal lobar degeneration
patients with ubiquitin pathology. GRN transcript haploinsufficiency is proposed as a …

[HTML][HTML] Discovery of small molecules that normalize the transcriptome and enhance cysteine cathepsin activity in progranulin-deficient microglia

MA Telpoukhovskaia, K Liu, FA Sayed, JI Etchegaray… - Scientific reports, 2020 - nature.com
Patients with frontotemporal dementia (FTD) resulting from granulin (GRN)
haploinsufficiency have reduced levels of progranulin and exhibit dysregulation in …

[HTML][HTML] Anti-sortilin1 Antibody Up-Regulates Progranulin via Sortilin1 Down-Regulation

S Miyakawa, H Sakuma, D Warude… - Frontiers in …, 2020 - frontiersin.org
Progranulin (PGRN) haploinsufficiency associated with loss-of-function mutations in the
granulin gene causes frontotemporal dementia (FTD). This suggests that increasing PGRN …

Opposing effects of progranulin deficiency on amyloid and tau pathologies via microglial TYROBP network

H Takahashi, ZA Klein, SM Bhagat, AC Kaufman… - Acta …, 2017 - Springer
Progranulin (PGRN) is implicated in Alzheimer's disease (AD) as well as frontotemporal
lobar degeneration. Genetic studies demonstrate an association of the common GRN …