Understanding unexpected large DNA changes from CRISPR in sickle cell
Deciphering unintended large gene modifications in gene editing for sickle cell disease
This project looks at why CRISPR gene editing can cause large, unexpected changes to the hemoglobin genes used to treat people with sickle cell disease.
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 ID | NIH-11124806 on NIH RePORTER |
What this research studies
Researchers are trying to understand why CRISPR editing of the hemoglobin genes can create large unintended DNA changes in blood stem cells taken from people with sickle cell disease. They have found large deletions and other rearrangements at the HBB, HBG, and BCL11A sites in patient-derived hematopoietic stem and progenitor cells. The team will track how long these changes persist, study the mechanisms that create them using patient cells and lab models, and test approaches to reduce these unwanted edits. You might be asked to donate blood or stem-cell samples to support this laboratory work.
Who could benefit from this research
Good fit: People with sickle cell disease who are willing to donate blood or hematopoietic stem/progenitor cell samples for laboratory research are the ideal participants for sample collection.
Not a fit: Patients seeking immediate clinical treatment or those who are not candidates for gene-editing approaches are unlikely to receive direct clinical benefit from this laboratory-focused work.
Why it matters
Potential benefit: If successful, this work could make gene-editing treatments for sickle cell disease safer by reducing harmful large-scale DNA changes.
How similar studies have performed: Some CRISPR-based therapies for sickle cell have shown clinical promise, but the issue of large unintended DNA changes is a recently recognized safety concern that remains unresolved.
Where this research is happening
Houston, United States
- Rice University — Houston, United States (Active)
Researchers
- Principal investigator: Bao, Gang — Rice University
- Study coordinator: Bao, Gang
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.