Understanding and reducing unintended large gene changes in sickle cell gene editing
Official title: Deciphering unintended large gene modifications in gene editing for sickle cell disease
Funded at Rice University · NIH-11386620 · Data updated 2026-11-12
A study in people with sickle cell disease looks at unexpected large DNA changes that can happen when repairing genes with CRISPR and aims to figure out how to prevent them.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Rice University NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project start | July 1, 2023 |
| Project end | June 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $626,816 |
| NIH project number | 5R01HL169761-04 |
| Project ID | NIH-11386620 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals with sickle cell disease who are candidates for gene-editing approaches using CRISPR/Cas9 in hematopoietic cells.
✕Less likely to help
People not undergoing gene-editing therapies or without access to patient-derived cells may not directly benefit.
What this research studies
This project examines how CRISPR/Cas9 editing in patients’ blood-forming cells can cause large, unintended changes in the genome near the target sickle cell genes. Researchers will analyze patient-derived cells to understand how these edits occur, what biological effects they have, and why some edits are riskier than others. The team will explore mechanisms behind large deletions and rearrangements and test strategies to minimize these unintended changes before therapies are used clinically. Findings could guide safer, more precise gene-editing approaches for sickle cell disease.
Why it matters
Potential benefit: If successful, the work could lead to safer gene-editing therapies for sickle cell disease by reducing harmful unintended genome alterations.
How similar studies have gone: Previous gene-editing trials show promise but have not fully addressed large unintended genome changes, making this a novel and important safety-focused advance.
Where this research is happening
-
Rice University
Houston, United States Active
Researchers
- Principal investigator: Bao, Gang — Rice University
- Study coordinator: Bao, Gang
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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.