Gene editing to turn on fetal hemoglobin for sickle cell disease
Novel therapeutic gene editing to induce fetal hemoglobin for sickle cell disease
The team edits a person's own blood stem cells with CRISPR to restart fetal hemoglobin production and help adults with sickle cell disease.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Jude Children's Research Hospital NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project ID | NIH-11166450 on NIH RePORTER |
What this research studies
Your own blood stem cells would be collected and edited with CRISPR/Cas9 to disrupt a switch so your red blood cells make fetal hemoglobin again. Lab work showed very high on-target editing and much more fetal hemoglobin in red cells, which greatly reduced sickling in preclinical tests. The researchers will refine the process, check thoroughly for unintended edits, and build the steps needed to bring this into patient treatment. If successful, the edited cells would be returned to your body to produce healthier red blood cells over time.
Who could benefit from this research
Good fit: Adults (age 21 and over) with sickle cell disease who are able to undergo stem cell collection and transplant procedures would be the ideal candidates.
Not a fit: Children under 21, people unable to tolerate stem cell mobilization/collection or transplant conditioning, or those with severe organ failure may not be eligible or likely to benefit.
Why it matters
Potential benefit: This could markedly reduce red blood cell sickling, pain crises, and organ damage and might offer a one-time curative option by increasing protective fetal hemoglobin.
How similar studies have performed: Other CRISPR-based editing of hematopoietic stem cells to raise fetal hemoglobin has produced promising early clinical results, though long-term outcomes remain under study.
Where this research is happening
Memphis, United States
- St. Jude Children's Research Hospital — Memphis, United States (Active)
Researchers
- Principal investigator: Weiss, Mitchell J — St. Jude Children's Research Hospital
- Study coordinator: Weiss, Mitchell J
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.