Developing a new test to evaluate gene editing treatments for sickle cell disease
Official title: A Modality-Agnostic Potency Assay Enabling Both Ex Vivo and In Vivo Genome Editing Therapeutics for Sickle Cell Disease
Funded at University of California Berkeley · NIH-11054587 · Data updated 2026-11-12
This study is working on a new lab test to check the quality of red blood cells made from gene editing treatments for sickle cell disease, so that patients can get the most effective care possible.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California Berkeley NIH-funded |
| Lab location | 1 site (Berkeley, United States) |
| Project start | April 28, 2023 |
| Project end | March 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $470,454 |
| NIH project number | 5U01AI176469-03 |
| Project ID | NIH-11054587 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with sickle cell disease who may benefit from gene editing therapies.
✕Less likely to help
Patients with other blood disorders or those who do not have sickle cell disease may not receive any benefit from this research.
What this research studies
This research focuses on creating a laboratory test that can accurately assess the quality of red blood cells (RBCs) derived from gene editing therapies for sickle cell disease (SCD). By utilizing advanced microfluidics technology, the study aims to develop a potency assay that can evaluate the biophysical properties of RBCs with high sensitivity and precision. This test will be crucial for determining the effectiveness of emerging gene editing treatments, ensuring that patients receive the best possible care. The research will involve both ex vivo (outside the body) and in vivo (inside the body) assessments to provide a comprehensive evaluation of these therapies.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene editing treatments for sickle cell disease, improving patient outcomes and quality of life.
How similar studies have gone: Previous research has shown promise in developing similar assays for other genetic disorders, indicating a potential for success in this novel approach.
Where this research is happening
-
University of California Berkeley
Berkeley, United States Active
Researchers
- Principal investigator: Giannikopoulos, Petros — University of California Berkeley
- Study coordinator: Giannikopoulos, Petros
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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.