New method to deliver gene editing tools for treating Sickle Cell Disease
Official title: Next generation of delivery platform for Prime editors based on an RNA vector for Sickle cell disease: the “all-in-one” vector
Funded at Mayo Clinic Rochester · NIH-11093962 · Data updated 2026-11-12
This study is exploring a new way to use a modified measles virus to help deliver gene editing tools that could fix the genetic issues causing Sickle Cell Disease, with the hope of improving treatment for patients like you.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mayo Clinic Rochester NIH-funded |
| Lab location | 1 site (Rochester, United States) |
| Project start | June 1, 2024 |
| Project end | April 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $181,575 |
| NIH project number | 5R21EB034346-02 |
| Project ID | NIH-11093962 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults with Sickle Cell Disease who are seeking innovative treatment options.
✕Less likely to help
Patients with Sickle Cell Disease who are under 21 years old or those who do not have the specific genetic mutations targeted by this research may not benefit.
What this research studies
This research focuses on developing a novel delivery system using a modified measles virus to effectively deliver gene editing tools for the treatment of Sickle Cell Disease. The approach aims to create a safe and efficient vector that can modify or correct specific genes without the need for traditional DNA templates. By expressing the Prime Editing system, the researchers hope to demonstrate the ability to correct genetic mutations in blood cells that cause Sickle Cell Disease, potentially leading to improved patient outcomes. The study will involve testing the functionality of this new delivery method in producing modified cells.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients with Sickle Cell Disease by correcting the underlying genetic mutations.
How similar studies have gone: While gene editing is a rapidly evolving field, this specific approach using a measles virus vector for Prime Editing is novel and has not been extensively tested in previous studies.
Where this research is happening
-
Mayo Clinic Rochester
Rochester, United States Active
Researchers
- Principal investigator: Devaux, Patricia — Mayo Clinic Rochester
- Study coordinator: Devaux, Patricia
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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.