NIH-funded research R01 grant

Investigating how specific brain cells contribute to abnormal movements in dystonia.

Official title: Cell Type Specific Genetic Manipulation to Dissect Cholinergic Interneuron Function and Plasticity in a Symptomatic Model of DYT1 Dystonia

Funded at Ut Southwestern Medical Center · NIH-11017050 · Data updated 2026-11-12

This study is looking at special brain cells that help control movement to understand why they don't work properly in people with DYT1 dystonia, a condition that causes twisting movements, and the goal is to find new ways to help those affected by similar movement issues.

More Dystonia research and trials

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUt Southwestern Medical Center
NIH-funded
Lab location1 site (Dallas, United States)
Project startFebruary 1, 2021
Project endJanuary 31, 2026
Funding year2025
Award (this year)$454,382
NIH project number5R01NS110853-05
Project IDNIH-11017050 on NIH RePORTER

Who could benefit from this research

Could help

Ideal candidates for this research are individuals diagnosed with DYT1 dystonia or those experiencing similar abnormal movements.

Less likely to help

Patients with movement disorders not related to cholinergic dysfunction may not benefit from this research.

What this research studies

This research focuses on cholinergic interneurons, which are important for controlling movement but are not well understood. The study aims to uncover how these neurons malfunction in conditions like DYT1 dystonia, a movement disorder. By using advanced genetic techniques in a mouse model, researchers will explore the cellular mechanisms that lead to abnormal twisting movements. The findings could help identify new treatment strategies for patients suffering from similar motor dysfunctions.

Why it matters

Potential benefit: If successful, this research could lead to improved treatments for patients with DYT1 dystonia and related movement disorders.

How similar studies have gone: Previous research has shown that targeting cholinergic neurons can lead to significant insights into movement disorders, suggesting this approach has potential for success.

Where this research is happening

  • Ut Southwestern Medical Center
    Dallas, United States
    Active

Researchers

  • Principal investigator: Dauer, William T. — Ut Southwestern Medical Center
  • Study coordinator: Dauer, William T.

Related research

Other NIH-funded projects on the same topics.

  • Role of THAP1 in brain cell support cells shaping brain development

    Aims to understand how the THAP1 protein in oligodendrocyte lineage cells helps shape the brain’s environment and myelination during development, which may relate to a movement disorder called DYT6 dystonia.

    NIH research Case Western Reserve University · Cleveland, United States · NIH-11596702

  • Leukocyte RNA biomarkers to help diagnose and monitor dystonia

    A small study will look for RNA signatures in blood that could help diagnose and track dystonia in people who have the condition.

    NIH research University of Tennessee Health Sci Ctr · Memphis, United States · NIH-11440474

  • How a dystonia-linked protein affects brain support cells and myelin

    This project looks at how the THAP1 protein linked to DYT6 dystonia changes the behavior of brain support cells and the surrounding matrix, which could affect myelination in adults with DYT-THAP1.

    NIH research Case Western Reserve University · Cleveland, United States · NIH-11418173

  • Intrathecal baclofen for children with dystonia due to brain injury

    A physician-led study at Baylor College of Medicine will help determine how ITB therapy affects daily life, comfort, and mobility for children with dystonia and related condition after brain injury.

    NIH research Baylor College of Medicine · Houston, United States · NIH-11401768

  • DYT1 Dystonia: Mouse Models to Understand TorsinA and Brain Circuits

    A researcher-led project uses mouse models to explore how a genetic change in DYT1 dystonia affects brain circuits and movement.

    NIH research University of Florida · Gainesville, United States · NIH-11399135

  • Dystonia-specific brain signaling pattern in cerebellum

    Aims to identify a characteristic pattern of nerve cell activity in the cerebellum that is linked to dystonia, with studies in mice and potential implications for people with the condition.

    NIH research Virginia Polytechnic Inst and St Univ · Blacksburg, United States · NIH-11398836

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.