Gene editing to raise fetal hemoglobin for sickle cell disease
Official title: Novel therapeutic gene editing to induce fetal hemoglobin for sickle cell disease
Funded at St. Jude Children's Research Hospital · NIH-11396296 · Data updated 2026-11-12
A new gene-editing therapy using patient’s own stem cells to boost fetal hemoglobin could potentially correct 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 start | September 1, 2023 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $1,183,000 |
| NIH project number | 5U01HL163983-04 |
| Project ID | NIH-11396296 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with sickle cell disease who are candidates for autologous stem cell therapies may be eligible.
✕Less likely to help
Patients whose disease is not driven by fetal hemoglobin or who cannot undergo stem cell procedures may not benefit.
What this research studies
This project aims to modify a patient’s hematopoietic stem cells so they produce more fetal hemoglobin, which can lessen the damage caused by sickle cell disease. The approach uses a precise CRISPR/Cas9 technique to disrupt a BCL11A binding motif, reactivating fetal hemoglobin production. The edited cells would be studied in labs and then tested in models to assess safety and how well they reduce red blood cell sickling, with the goal of translating findings to patients.
Why it matters
Potential benefit: If successful, this therapy could offer a potentially curative option for sickle cell disease by reducing disease symptoms and improving quality of life.
How similar studies have gone: Previous work showing high on-target editing and HbF production in edited cells supports progression, though this specific clinical translation is ongoing.
Where this research is happening
-
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
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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.