NIH-funded research R01 grant

Understanding the role of UBQLN2 in ALS and frontotemporal dementia

Official title: Investigation of UBQLN2 in neuronal dysfunction and ALS-FTD

Funded at University of Colorado · NIH-11098689 · Data updated 2026-11-12

This study is looking at how changes in the UBQLN2 gene might cause problems in the brain for people with ALS-FTD, with the hope of finding new ways to help treat those affected by this condition.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of Colorado
NIH-funded
Lab location1 site (Boulder, UNITED STATES)
Project startJune 15, 2023
Project endMay 31, 2028
Funding year2025
Award (this year)$650,937
NIH project number5R01NS131660-03
Project IDNIH-11098689 on NIH RePORTER

Who could benefit from this research

Could help

Ideal candidates for this research include individuals diagnosed with ALS-FTD, particularly those with a familial history of the disease.

Less likely to help

Patients with other forms of neurodegenerative diseases unrelated to ALS-FTD may not benefit from this research.

What this research studies

This research investigates the role of the UBQLN2 gene in the neurodegenerative disease known as Amyotrophic Lateral Sclerosis with frontotemporal dementia (ALS-FTD). The study aims to uncover the mechanisms by which mutations in UBQLN2 contribute to the disease, focusing on how it affects the degradation of problematic proteins in neurons. By examining both familial and sporadic cases of ALS-FTD, the research seeks to identify potential therapeutic targets that could lead to effective treatments for patients suffering from this condition.

Why it matters

Potential benefit: If successful, this research could lead to new treatments that significantly improve the quality of life and longevity for patients with ALS-FTD.

How similar studies have gone: Previous research has shown promise in understanding the genetic factors contributing to ALS-FTD, suggesting that this approach could yield valuable insights.

Where this research is happening

  • University of Colorado
    Boulder, United States
    Active

Researchers

  • Principal investigator: Whiteley, Alexandra — University of Colorado
  • Study coordinator: Whiteley, Alexandra

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.
Last reviewed 2026-11-14 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.