Investigating genetic changes that affect how platelets function in blood.
Official title: Evaluation of genetic variants affecting platelet function with CRISPR HDR in human megakaryocytes
Funded at University of Utah · NIH-10917269 · Data updated 2026-11-12
This study is looking at how certain genetic changes affect how well your platelets work, especially for people with bleeding disorders, and it uses advanced technology to make specific changes in the cells that create platelets to help find better treatments for these conditions.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Utah NIH-funded |
| Lab location | 1 site (Salt Lake City, United States) |
| Project start | September 1, 2023 |
| Project end | June 30, 2028 |
| Funding year | 2024 |
| Award (this year) | $526,252 |
| NIH project number | 5R01HL166805-02 |
| Project ID | NIH-10917269 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals with known or suspected inherited platelet function disorders, particularly those under 21 years old.
✕Less likely to help
Patients without any genetic variants affecting platelet function or those with non-inherited bleeding disorders may not benefit from this research.
What this research studies
This research focuses on understanding genetic variants that impact platelet function, particularly those linked to bleeding disorders. Using a cutting-edge CRISPR technology, the study aims to make precise genetic modifications in megakaryocytes, the cells that produce platelets. By evaluating these changes, researchers hope to clarify the significance of various genetic variants that have previously been classified as unknown. This could lead to better understanding and treatment options for patients with platelet-related disorders.
Why it matters
Potential benefit: If successful, this research could lead to improved diagnosis and treatment for patients with inherited bleeding disorders.
How similar studies have gone: Other research using CRISPR technology for genetic modifications in blood cells has shown promising results, indicating potential success for this approach.
Where this research is happening
-
University of Utah
Salt Lake City, United States Active
Researchers
- Principal investigator: Rowley, Jesse — University of Utah
- Study coordinator: Rowley, Jesse
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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.