Targeted lysosomal RNA degradation for disease-relevant RNAs
Official title: Targeted lysosomal degradation of RNA
Funded at Tufts University Medford · NIH-11445383 · Data updated 2026-11-12
A new approach uses tiny molecules to tag specific RNAs for destruction inside cells, with the goal of helping diseases linked to RNA problems.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Tufts University Medford NIH-funded |
| Lab location | 1 site (Boston, UNITED STATES) |
| Project start | August 1, 2026 |
| Project end | July 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $374,866 |
| NIH project number | 1R21TR006446-01 |
| Project ID | NIH-11445383 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal participants would be individuals whose disease involves RNA-guided pathology and could plausibly benefit from RNA-targeting therapies in future development.
✕Less likely to help
Patients with conditions unlikely to be affected by RNA-targeting therapies or those without access to future trials may not benefit.
What this research studies
This project proposes a novel way to remove harmful RNA molecules by guiding them to the cell’s lysosome for breakdown. It builds on recent RNA-binding molecules and combines them with the cell’s degradation machinery to create a therapeutic strategy. By targeting RNA via autophagy (RNAphagy), the work aims to affect RNAs involved in disease while aiming to minimize harming healthy cells. The R21 study will explore safety, feasibility, and initial signals of efficacy in cellular and potentially animal models. If successful, this could open a new class of RNA-focused treatments.
Why it matters
Potential benefit: If successful, patients could benefit from new RNA-targeting therapies that reduce the harmful effects of disease-related RNAs.
How similar studies have gone: Preliminary work shows RNA-targeting approaches can work for specific RNAs, but broad, lysosome-based RNA degradation is a novel direction with early, promising signals.
Where this research is happening
-
Tufts University Medford
Boston, United States Active
Researchers
- Principal investigator: Kritzer, Joshua a — Tufts University Medford
- Study coordinator: Kritzer, Joshua a
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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.