RNA therapy that targets APP for Alzheimer's disease
Treating Alzheimer's Disease with PMO RNA Therapeutics
This project develops an RNA-based medicine to lower the APP protein that leads to toxic Aβ42 and could help people with Alzheimer's disease.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-11237558 on NIH RePORTER |
What this research studies
Researchers are designing PMO antisense RNA molecules that bind the APP message and reduce production of the Aβ42 peptide that forms plaques. They will test these molecules in lab-grown cells and animal models of Alzheimer's to see if they lower Aβ42, reduce plaque and tangle formation, and protect neurons. A major part of the work focuses on improving delivery into brain cells so the drug can reach its target. If the laboratory and animal tests look promising, the team aims to move the approach toward human testing.
Who could benefit from this research
Good fit: People with early-stage Alzheimer's disease or mild cognitive impairment with evidence of amyloid pathology would be the most likely candidates for future trials.
Not a fit: People with non-Alzheimer's dementias or very advanced Alzheimer's with extensive neuron loss are unlikely to benefit from this APP-targeting approach.
Why it matters
Potential benefit: If successful, this approach could reduce the root-cause protein production (APP/Aβ42) and potentially slow or prevent cognitive decline in people with Alzheimer's.
How similar studies have performed: RNA therapeutics, including PMO antisense drugs, have been FDA-approved for other diseases and shown success in nervous system disorders, but targeting APP in Alzheimer's is a novel and early-stage application.
Where this research is happening
La Jolla, United States
- University of California, San Diego — La Jolla, United States (Active)
Researchers
- Principal investigator: Dowdy, Steven F — University of California, San Diego
- Study coordinator: Dowdy, Steven F
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.