Shortened TDP‑43 proteins in ALS and frontotemporal dementia
Regulation and impact of shortened TDP43 isoforms in FTD and ALS
['FUNDING_R37'] · UNIVERSITY OF MICHIGAN AT ANN ARBOR · NIH-11225145
This project studies whether shortened forms of the TDP‑43 protein cause or worsen damage in people with ALS and frontotemporal dementia to guide future treatments.
Quick facts
| Phase | ['FUNDING_R37'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF MICHIGAN AT ANN ARBOR (nih funded) |
| Locations | 1 site (ANN ARBOR, UNITED STATES) |
| Trial ID | NIH-11225145 on ClinicalTrials.gov |
What this research studies
From your perspective, researchers want to know why shortened (sTDP43) versions of the TDP‑43 protein build up in nerve cells and how that leads to the clumping seen in ALS and frontotemporal dementia. They will use overactive neuron models in the lab, molecular RNA and protein techniques, and comparisons with human tissue to track how sTDP43 forms and affects cells. The team combines RNA and protein biology approaches to see how sTDP43 disrupts normal TDP‑43 function and RNA handling. The goal is to spot pathways or markers that could become targets for new treatments or earlier diagnosis.
Who could benefit from this research
Good fit: Ideal candidates would be people with ALS or frontotemporal dementia who might donate tissue samples, join related observational efforts, or become eligible for future therapies targeting TDP‑43.
Not a fit: People without ALS or frontotemporal dementia, or whose illness is not driven by TDP‑43 changes, are unlikely to get direct benefit from this research.
Why it matters
Potential benefit: If successful, this work could reveal new targets to stop or slow TDP‑43 damage and lead to treatments or tests for people with ALS and frontotemporal dementia.
How similar studies have performed: Prior laboratory work showed shortened TDP‑43 can aggregate in overactive neurons and recreate key disease features, but applying those findings to patients is still early and not yet proven.
Where this research is happening
ANN ARBOR, UNITED STATES
- UNIVERSITY OF MICHIGAN AT ANN ARBOR — ANN ARBOR, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BARMADA, SAMI — UNIVERSITY OF MICHIGAN AT ANN ARBOR
- Study coordinator: BARMADA, SAMI
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions: Amyotrophic Lateral Sclerosis Motor Neuron Disease