Gene editing to raise fetal hemoglobin levels in adults with 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-11378801 · Data updated 2026-11-12
This project edits a patient’s own blood stem cells with CRISPR to turn on fetal hemoglobin and help adults with sickle cell disease have fewer sickled red cells and painful episodes.
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 | 2025 |
| Award (this year) | $185,000 |
| NIH project number | 3U01HL163983-03S1 |
| Project ID | NIH-11378801 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with sickle cell disease (age 21+) who are eligible for autologous stem cell collection and the conditioning/transplant procedures would be the intended candidates.
✕Less likely to help
People under 21, or those who cannot tolerate stem cell collection or transplant conditioning because of other medical issues, would likely not be eligible or benefit.
What this research studies
If I take part, doctors would collect my blood-forming stem cells, use a CRISPR/Cas9 ribonucleoprotein to disrupt a BCL11A-related switch, and return my edited cells so my red blood cells make more fetal hemoglobin. In lab and mouse-transplant tests, the team achieved high on-target editing in long-term stem cells with no off-target mutations detected at high sensitivity and produced red cells with much higher HbF and far less sickling. The group plans to move this work into clinical use, with careful monitoring of blood counts, HbF levels, and safety after transplant. Treatment will involve specialized procedures and follow-up over months to years to track durability and side effects.
Why it matters
Potential benefit: If successful, this could produce long-lasting increases in fetal hemoglobin that reduce or eliminate red-cell sickling, leading to fewer pain crises and improved organ health.
How similar studies have gone: Related gene-editing approaches targeting BCL11A or HbF induction have shown promising early clinical results, with some patients achieving major reductions in symptoms and transfusion needs.
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.