Investigating genetic changes that affect how platelets function in blood.
Evaluation of genetic variants affecting platelet function with CRISPR HDR in human megakaryocytes
['FUNDING_R01'] · UNIVERSITY OF UTAH · NIH-10917269
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
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF UTAH (nih funded) |
| Locations | 1 site (SALT LAKE CITY, UNITED STATES) |
| Trial ID | NIH-10917269 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals with known or suspected inherited platelet function disorders, particularly those under 21 years old.
Not a fit: Patients without any genetic variants affecting platelet function or those with non-inherited bleeding disorders may not benefit from this research.
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 performed: 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
SALT LAKE CITY, UNITED STATES
- UNIVERSITY OF UTAH — SALT LAKE CITY, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: ROWLEY, JESSE — UNIVERSITY OF UTAH
- Study coordinator: ROWLEY, JESSE
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 Coagulation Disorders, Blood Platelet Disorders