TDP-43 and misplaced neuron messages in ALS

Mechanistic analysis of TDP-43-mediated RNA localization in neurons and its misregulation in ALS

NIH-funded research University of Colorado Denver · NIH-11168725

Researchers will map how the protein TDP-43 causes RNA messages inside nerve cells to end up in the wrong places in people with ALS.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of Colorado Denver NIH-funded
Lab location1 site (Aurora, UNITED STATES)
Project IDNIH-11168725 on NIH RePORTER

What this research studies

This project uses patient-derived or lab-grown nerve cells to see where RNA messages travel inside long nerve fibers, focusing on the TDP-43 protein linked to ALS. The team combines stem cell-based neuron models, live-cell microscopy, and subcellular transcriptomics to read which RNAs are present in different parts of the cell. They developed a new method to detect RNA misplacement across the whole transcriptome and will test how TDP-43 recognizes and moves these RNAs. The investigators aim to connect specific mislocalized RNAs to changes in neuron function that may drive ALS.

Who could benefit from this research

Good fit: Ideal candidates would be people with ALS who are willing to donate blood or skin samples for creation of stem-cell models or who want to be contacted about related clinical studies.

Not a fit: People without ALS or those seeking an immediate therapy are unlikely to receive direct benefit from this laboratory-focused research.

Why it matters

Potential benefit: If successful, this work could reveal new molecular targets to prevent or slow nerve-cell damage in ALS.

How similar studies have performed: Prior research has linked TDP-43 to ALS and shown some RNA mislocalization, but using transcriptome-wide live-cell tracking of RNA localization is a novel approach.

Where this research is happening

Aurora, 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.
Conditions Amyotrophic Lateral Sclerosis Motor Neuron DiseaseAran-Duchenne disease
Last reviewed 2026-06-13 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.