Improving how oligonucleotide therapies are delivered to treat diseases.
Official title: Accelerating the translation of oligonucleotide therapeutics by enhancing delivery
Funded at University of California-Irvine · NIH-11132147 · Data updated 2026-11-12
This study is working on better ways to deliver new treatments called oligonucleotide therapeutics, which could help people with tough conditions like spinal muscular atrophy and amyloidosis, making these treatments more effective for patients.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California-Irvine NIH-funded |
| Lab location | 1 site (Irvine, United States) |
| Project start | June 16, 2025 |
| Project end | June 15, 2027 |
| Funding year | 2025 |
| Award (this year) | $419,398 |
| NIH project number | 1R21TR005645-01 |
| Project ID | NIH-11132147 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include patients with conditions that could be treated with oligonucleotide therapies, such as spinal muscular atrophy or hATTR amyloidosis.
✕Less likely to help
Patients with conditions that do not involve oligonucleotide therapeutics or those who are not responsive to such treatments may not benefit from this research.
What this research studies
This research focuses on enhancing the delivery mechanisms of oligonucleotide therapeutics (ONTs), which are promising treatments for various diseases, including those previously deemed untreatable. The project aims to overcome the significant barriers that ONTs face in reaching their intended targets within the body, particularly due to their large size and charged nature. By developing new delivery strategies, the research seeks to improve the effectiveness of these therapies, potentially leading to better patient outcomes. Patients may benefit from advancements in treatments for conditions like spinal muscular atrophy and amyloidosis.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for a range of serious diseases, improving patient health and quality of life.
How similar studies have gone: Other research has shown promise in enhancing the delivery of oligonucleotide therapies, indicating that this approach could lead to significant advancements in treatment efficacy.
Where this research is happening
-
University of California-Irvine
Irvine, United States Active
Researchers
- Principal investigator: Edinger, Aimee L — University of California-Irvine
- Study coordinator: Edinger, Aimee L
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.