Gene-editing delivery for sickle cell disease using safer, non-viral nanoparticles
Official title: Therapeutic gene editing nanoparticles for sickle cell disease
Funded at University of Michigan at Ann Arbor · NIH-11451335 · Data updated 2026-11-12
A researcher is developing a safer, non-viral nanoparticle method to deliver gene edits to a patient’s own blood-forming cells to treat sickle cell disease.
Quick facts
| Grant type | Career grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Michigan at Ann Arbor NIH-funded |
| Lab location | 1 site (Ann Arbor, United States) |
| Project start | September 1, 2026 |
| Project end | May 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $167,400 |
| NIH project number | 1K08HL175254-01A1 |
| Project ID | NIH-11451335 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with sickle cell disease who lack matched donors or who may benefit from autologous (self) gene-edited cells.
✕Less likely to help
Patients whose disease is not driven by the sickle cell mutation or who cannot undergo stem cell procedures may not benefit.
What this research studies
This program focuses on creating non-viral delivery systems to genetically modify a patient’s hematopoietic stem cells, aiming to reduce toxicity from conditioning and improve engraftment. The project will explore how nanoparticle delivery can correct the sickle cell mutation with fewer side effects than current viral methods. It involves collaboration with a clinical team to translate the approach toward real patients who have limited donor options or access to matched transplants.
Why it matters
Potential benefit: If successful, patients with sickle cell disease could receive a personalized gene correction with less toxicity and broader access.
How similar studies have gone: Prior gene-editing efforts show promise, but non-viral, lower-toxicity delivery remains a developing area.
Where this research is happening
-
University of Michigan at Ann Arbor
Ann Arbor, United States Active
Researchers
- Principal investigator: Triebwasser, Michael Paul — University of Michigan at Ann Arbor
- Study coordinator: Triebwasser, Michael Paul
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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.