Understanding unintended gene changes in gene editing for sickle cell disease
Official title: Deciphering unintended large gene modifications in gene editing for sickle cell disease
Funded at Rice University · NIH-10876342 · Data updated 2026-11-12
This study is looking into how gene editing for sickle cell disease might accidentally change more than just the intended genes in patients' blood cells, and it aims to help make these treatments safer and more effective for people with the condition.
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 | 2024 |
| Award (this year) | $632,319 |
| NIH project number | 5R01HL169761-02 |
| Project ID | NIH-10876342 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with sickle cell disease who are considering or undergoing gene editing therapies.
✕Less likely to help
Patients with sickle cell disease who are not eligible for gene editing therapies or those who have not been diagnosed may not benefit from this research.
What this research studies
This research investigates the unintended large gene modifications that can occur during CRISPR gene editing for sickle cell disease (SCD). It focuses on how these modifications, such as large deletions and insertions, happen at the targeted gene sites in patients' blood cells. By studying these effects, the research aims to uncover the biological consequences and mechanisms behind these changes, which are currently not well understood. The findings could lead to improved safety and efficacy of gene editing therapies for SCD patients.
Why it matters
Potential benefit: If successful, this research could enhance the safety of gene editing treatments for sickle cell disease, potentially leading to better patient outcomes.
How similar studies have gone: Other research has shown promise in using CRISPR for gene editing in sickle cell disease, but the specific focus on unintended large gene modifications is a novel approach.
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.