New method to deliver gene editing tools for treating Sickle Cell Disease
Next generation of delivery platform for Prime editors based on an RNA vector for Sickle cell disease: the “all-in-one” vector
['FUNDING_R21'] · MAYO CLINIC ROCHESTER · NIH-11093962
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
| Phase | ['FUNDING_R21'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | MAYO CLINIC ROCHESTER (nih funded) |
| Locations | 1 site (ROCHESTER, UNITED STATES) |
| Trial ID | NIH-11093962 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults with Sickle Cell Disease who are seeking innovative treatment options.
Not a fit: 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.
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 performed: 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
ROCHESTER, UNITED STATES
- MAYO CLINIC ROCHESTER — ROCHESTER, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: DEVAUX, PATRICIA — MAYO CLINIC ROCHESTER
- Study coordinator: DEVAUX, PATRICIA
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 below.
Conditions: Blood Diseases, blood disorder