TDP-43 protein and 'jumping genes' in Alzheimer's and related dementias
Effects of TDP-43 Proteinopathy on Retrotransposon Activation and Cell-Type Specific Vulnerability in a Mammalian Model of Alzheimer's and Related Dementias
This project looks at whether a damaged brain protein called TDP-43 and reactivated 'jumping genes' cause harm to brain cells in people with Alzheimer's and related dementias.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | State University New York Stony Brook NIH-funded |
| Lab location | 1 site (Stony Brook, United States) |
| Project ID | NIH-11242008 on NIH RePORTER |
What this research studies
Researchers will use specially engineered mice that carry a human TDP-43 mutation linked to dementia to see how TDP-43 affects 'jumping genes' (retrotransposons) in neurons and support cells. They will also use targeted viral tools to increase TDP-43 only in neurons or only in astrocytes to watch whether harm spreads between cell types. The team will measure retrotransposon activity, cell loss, and molecular signs of toxicity to determine how these events relate to neurodegeneration. The project establishes a first mammalian platform to connect TDP-43, retrotransposons, and dementia processes.
Who could benefit from this research
Good fit: People with Alzheimer's disease or related dementias, especially those whose illness shows signs of TDP-43 pathology, would be most relevant to future clinical work building on these findings.
Not a fit: Patients whose dementia is driven by unrelated mechanisms or who need immediate clinical treatment are unlikely to gain direct benefit from this lab-based mouse research.
Why it matters
Potential benefit: If successful, this work could reveal new targets (TDP-43 or retrotransposon activity) for treatments aimed at slowing or preventing neuron loss in Alzheimer's and related dementias.
How similar studies have performed: TDP-43 has been linked to ALS and frontotemporal dementia and retrotransposon involvement is an emerging idea, but applying a mammalian TDP-43 model to test retrotransposon-driven neurodegeneration is a novel approach.
Where this research is happening
Stony Brook, United States
- State University New York Stony Brook — Stony Brook, United States (Active)
Researchers
- Principal investigator: Sher, Roger B — State University New York Stony Brook
- Study coordinator: Sher, Roger B
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.