Gene editing in bone marrow to treat blood disorders
Official title: Precise in vivo gene editing of HSPC for the treatment of genetic hematologic diseases
Funded at University of Kentucky · NIH-10756130 · Data updated 2026-11-12
This study is exploring a new way to use gene editing to treat blood disorders like sickle cell disease and β-thalassemia by making changes directly in the bone marrow, which could be safer and cheaper than current methods.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Kentucky NIH-funded |
| Lab location | 1 site (Lexington, United States) |
| Project start | January 1, 2023 |
| Project end | December 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $191,250 |
| NIH project number | 5R21HL166178-02 |
| Project ID | NIH-10756130 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with genetic blood disorders such as sickle cell disease or β-thalassemia.
✕Less likely to help
Patients with non-genetic blood disorders or those who do not have hematologic conditions may not benefit from this research.
What this research studies
This research investigates a new method of gene editing using CRISPR/cas9 technology directly in the bone marrow to treat genetic blood disorders like sickle cell disease and β-thalassemia. Instead of removing stem cells from patients for editing, the approach aims to edit these cells in place, which could reduce risks and costs associated with traditional methods. The researchers are developing a novel delivery system that uses baculoviral vectors combined with magnetic nanoparticles to enhance the precision and effectiveness of gene editing. This innovative technique seeks to overcome challenges related to off-target effects and low engraftment efficiency seen in current therapies.
Why it matters
Potential benefit: If successful, this research could provide a safer and more efficient treatment option for patients with genetic hematologic diseases.
How similar studies have gone: While gene editing has shown promise in other contexts, this specific in vivo approach using baculoviral vectors is relatively novel and has not been extensively tested in clinical settings.
Where this research is happening
-
University of Kentucky
Lexington, United States Active
Researchers
- Principal investigator: Tong, Sheng — University of Kentucky
- Study coordinator: Tong, Sheng
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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.