Investigating how a specific TDP43 protein variant contributes to neurodegeneration in FTD and ALS

Atypical TDP43 isoforrms driving neurodegeneration in FTD/ALS

['FUNDING_R01'] · UNIVERSITY OF MICHIGAN AT ANN ARBOR · NIH-10747395

This study is looking at a special version of a protein called TDP43 to see how it affects brain cells in people with frontotemporal dementia and ALS, with the goal of finding new ways to help improve their condition.

Quick facts

Phase['FUNDING_R01']
Study typeNih_funding
SexAll
SponsorUNIVERSITY OF MICHIGAN AT ANN ARBOR (nih funded)
Locations1 site (ANN ARBOR, UNITED STATES)
Trial IDNIH-10747395 on ClinicalTrials.gov

What this research studies

This research focuses on understanding the role of a unique variant of the TDP43 protein in the development of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). It aims to explore how this variant, which is mislocalized within neurons, contributes to neuronal hyperexcitability and neurodegeneration. By examining the relationship between this TDP43 variant and neuronal activity, the researchers hope to uncover new therapeutic strategies that could restore normal TDP43 function and improve patient outcomes. The study employs both in vitro and in vivo models to investigate these mechanisms.

Who could benefit from this research

Good fit: Ideal candidates for this research are individuals diagnosed with frontotemporal dementia or amyotrophic lateral sclerosis.

Not a fit: Patients with other forms of dementia or neurodegenerative diseases unrelated to TDP43 pathology may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to new treatments that slow down or prevent the progression of FTD and ALS.

How similar studies have performed: Previous research has shown promising results in understanding TDP43 pathology, indicating potential for breakthroughs in treatment approaches.

Where this research is happening

ANN ARBOR, 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: Amyotrophic Lateral Sclerosis Motor Neuron Disease, Gehrig's Disease, Lou Gehrig Disease

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.