RNAi-based approach to modify ApoE, immune signals, and tau for a potential Alzheimer’s treatment
Official title: RNAi-Mediated Combinatorial Modulation of ApoE, Innate Immune Response, and Tau Pathology for a Disease-Modifying Treatment of Alzheimer's Disease (AD)
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11300816 · Data updated 2026-11-12
A gene-silencing strategy in the brain aims to slow or alter Alzheimer’s disease by targeting ApoE, immune signals, and tau together.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project start | September 1, 2026 |
| Project end | June 30, 2031 |
| Funding year | 2026 |
| Award (this year) | $419,946 |
| NIH project number | 1R01AG099051-01 |
| Project ID | NIH-11300816 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with or at risk for Alzheimer’s disease who are eligible for CNS-targeted gene-silencing approaches in future trials.
✕Less likely to help
Patients unlikely to benefit are those without Alzheimer’s pathology or those not suited for CNS-delivery therapies.
What this research studies
This project will use CNS-active multi-valent siRNA to temporarily silence multiple genes involved in ApoE, immune response, and tau pathology in animal models and, later, in humans. The work will help define how each pathway contributes to disease progression and whether their combined modulation can more effectively slow neurodegeneration. Researchers plan to optimize the siRNA scaffolds for long-lasting brain activity with minimal systemic exposure and prepare for upcoming clinical trials. Results could guide future therapies that target several AD drivers at once.
Why it matters
Potential benefit: If successful, this approach could lead to a disease-modifying treatment that slows progression by targeting multiple AD drivers.
How similar studies have gone: Preclinical studies show that multi-gene RNAi can silence CNS targets for months and impact disease pathways.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Khvorova, Anastasia — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Khvorova, Anastasia
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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.