NIH-funded research R01 grant

Harnessing neuron-derived vesicles to find Huntington’s disease biomarkers

Official title: Harnessing neuronal extracellular vesicles for Huntington disease biomarkers

Funded at University of Central Florida · NIH-11448886 · Data updated 2026-11-12

A research project aims to identify brain-derived biomarkers in blood that reflect Huntington disease activity, using neuronal extracellular vesicles as the signal source.

More Huntington Disease research and trials

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of Central Florida
NIH-funded
Lab location1 site (Orlando, United States)
Project startSeptember 15, 2026
Project endMay 31, 2031
Funding year2026
Award (this year)$545,462
NIH project number1R01NS144755-01A1
Project IDNIH-11448886 on NIH RePORTER

Who could benefit from this research

Could help

Individuals with Huntington disease or at-risk family members who may participate in biomarker validation studies or future trials.

Less likely to help

There may be no direct benefit for participants if the biomarkers do not correlate with disease activity or if tests are not adopted clinically.

What this research studies

This project looks for brain-specific markers in blood by studying extracellular vesicles that come from neurons. It focuses on Huntington disease and how mutant HTT signals might be measured in a less invasive way than current spinal fluid tests. The team will analyze how well these vesicles carry HTT-related information and how stable and detectable these signals are in people or model systems. Findings could help track disease progression or response to therapies that lower HTT or reduce somatic HTT expansion. The work is conducted at the University of Central Florida and led by Amber L. Southwell.

Why it matters

Potential benefit: If successful, patients could benefit from a simpler, less invasive way to monitor disease progression and treatment effects.

How similar studies have gone: Previous studies have shown that brain-derived extracellular vesicles can carry disease-relevant signals, supporting this approach as promising but still needing validation.

Where this research is happening

  • University of Central Florida
    Orlando, United States
    Active

Researchers

  • Principal investigator: Southwell, Amber L — University of Central Florida
  • Study coordinator: Southwell, Amber L

Related research

Other NIH-funded projects on the same topics.

  • Understanding how blood flow problems in the brain affect Huntington’s disease

    A study at a major hospital is looking at how blood vessels in the brain may contribute to symptoms and brain changes in Huntington’s disease.

    NIH research Massachusetts General Hospital · Boston, United States · NIH-11371125

  • Rhes‑SUMO pathway's role in Huntington's disease

    This work looks at whether the Rhes‑SUMO protein system increases toxic huntingtin and helps it spread in the brain, which may explain early striatal damage in people with Huntington's disease.

    NIH research Florida Atlantic University · Boca Raton, United States · NIH-11299742

  • Understanding how ribosome stalling affects Huntington disease

    This study is looking at how a problem with protein production in the brain might contribute to Huntington disease, which could help us find new ways to treat the condition.

    NIH research Florida Atlantic University · Boca Raton, United States · NIH-11264540

  • Rhes-SUMO Pathway and Huntington Disease in Mice

    A study in mouse models explores how the Rhes-SUMO pathway may drive brain and nerve cell vulnerability in Huntington disease.

    NIH research Florida Atlantic University · Boca Raton, United States · NIH-11176921

  • Investigating the role of the Rhes-SUMO pathway in Huntington's disease

    This study is looking at how a specific pathway in the brain might affect the worsening of Huntington's disease, with the hope of finding new ways to help manage the symptoms and slow down the disease for those living with it.

    NIH research University of Florida · Gainesville, United States · NIH-11175740

  • Understanding how Huntington disease disrupts protein production in brain cells

    Researchers are studying Huntington disease to learn how a faulty protein blocks ribosomes and slows protein-making in brain cells, which may contribute to movement and thinking problems.

    NIH research Florida Atlantic University · Boca Raton, United States · NIH-11126038

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.