Increasing progranulin levels in a brain model of Frontotemporal Dementia

Upregulation of progranulin in a human iPSC-derived neurovascular model of GRN-associated Frontotemporal Dementia

['FUNDING_R21'] · UNIV OF MASSACHUSETTS MED SCH WORCESTER · NIH-10789724

This study is looking at a protein called progranulin that’s connected to Frontotemporal Dementia (FTD) to better understand how different brain cells are involved in the disease and to find ways to boost progranulin levels, which could lead to new treatments that help people with FTD.

Quick facts

Phase['FUNDING_R21']
Study typeNih_funding
SexAll
SponsorUNIV OF MASSACHUSETTS MED SCH WORCESTER (nih funded)
Locations1 site (WORCESTER, UNITED STATES)
Trial IDNIH-10789724 on ClinicalTrials.gov

What this research studies

This research investigates the role of progranulin, a protein linked to Frontotemporal Dementia (FTD), in a human brain model created from induced pluripotent stem cells. The study aims to understand how different cell types contribute to the disease and to explore a method for increasing progranulin levels in these cells. By focusing on the blood-brain barrier and its associated cells, the research seeks to identify new therapeutic strategies that could help mitigate the effects of FTD. Patients may benefit from insights gained about the disease mechanisms and potential treatments.

Who could benefit from this research

Good fit: Ideal candidates for this research are individuals with familial Frontotemporal Dementia, particularly those with known mutations in the progranulin gene.

Not a fit: Patients with non-genetic forms of dementia or those without a family history of Frontotemporal Dementia may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to new therapies that improve outcomes for patients with Frontotemporal Dementia.

How similar studies have performed: Previous research has shown promise in targeting progranulin levels in neurodegenerative diseases, suggesting that this approach may yield beneficial results.

Where this research is happening

WORCESTER, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Conditions: Alzheimer disease dementia, Alzheimer syndrome

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.