Last reviewed: June 1, 2026
2017Molecular Neurodegeneration
Lysosomal processing of progranulin
Zhou X, Paushter DH, Feng T, Sun L, et al.
Abstract summary
Showed that progranulin localizes to lysosomes in microglia and regulates lysosomal cathepsin activity and pH. GRN-deficient microglia have enlarged lysosomes with aberrant cathepsin levels. This lysosomal dysfunction phenotype can be rescued by progranulin supplementation, the basis for recombinant GRN therapy approaches.
Evidence labels
in vitro assayanimal modelhuman tissue
Targets
Diseases
- Frontotemporal dementia
Species
mouse, human
Methods
cell biology, organelle fractionation, live imaging
Therapeutic relevance
Establishes the lysosomal GRN pathway as the direct therapeutic target for progranulin replacement in FTD.
Last reviewed: June 1, 2026