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Updated: Feb 9, 2026
09:17
Using Retinal Imaging to Study Dementia
Published on: November 6, 2017
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Progranulin as a therapeutic target for dementia
Daniela Galimberti1, Chiara Fenoglio1, Elio Scarpini1
1a Neurodegenerative Diseases Unit, University of Milan, Centro Dino Ferrari, Fondazione Cà Granda, IRCCS Ospedale Maggiore Policlinico , Milan , Italy.
Expert Opinion on Therapeutic Targets
|June 12, 2018
Summary
Progranulin (PGRN) gene mutations cause frontotemporal dementia (FTD). Measuring plasma PGRN levels can identify carriers before symptoms appear, offering a potential therapeutic target for FTD.
Area of Science:
- Neuroscience
- Genetics
- Biochemistry
Background:
- Progranulin (PGRN) is synthesized during spermatogenesis and has anti-inflammatory properties.
- Mutations in the PGRN gene (GRN) cause frontotemporal dementia (FTD) via haploinsufficiency.
- Low plasma PGRN levels are a biomarker for GRN mutation carriers.
Purpose of the Study:
- To review current knowledge on PGRN functions and GRN mutations in FTD.
- To summarize FTD clinical trials and PGRN-replacement strategies.
- To discuss PGRN as a therapeutic target for FTD.
Main Methods:
- Literature review of PGRN physiology, GRN mutations, and FTD.
- Analysis of clinical trials and preclinical PGRN-replacement models.
- Evaluation of PGRN's therapeutic potential and associated risks.
Main Results:
- GRN mutations leading to PGRN haploinsufficiency are linked to familial FTD.
- Plasma PGRN levels are a reliable biomarker for identifying mutation carriers.
- PGRN is a promising therapeutic target for FTD, especially in preclinical stages.
Conclusions:
- PGRN is a promising therapeutic target for frontotemporal dementia.
- Early intervention in preclinical stages is ideal for targeting pathogenic mechanisms.
- Potential side effects like tumorigenesis and lack of outcome measures complicate PGRN-based therapies.
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