New gene editing approach to increase 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-10917350 · Data updated 2026-11-12
This study is testing a new treatment for sickle cell disease that uses gene editing to help your body make healthier red blood cells, which could help reduce or even get rid of your symptoms.
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 | 2024 |
| Award (this year) | $1,159,340 |
| NIH project number | 5U01HL163983-02 |
| Project ID | NIH-10917350 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 21 years old or younger.
✕Less likely to help
Patients with other blood disorders or those who do not have sickle cell disease may not benefit from this research.
What this research studies
This research focuses on developing a groundbreaking therapy for sickle cell disease by using gene editing techniques to modify a patient's own blood stem cells. The goal is to enhance the production of fetal hemoglobin, which can significantly reduce or eliminate the symptoms of the disease. By utilizing a method called CRISPR, researchers aim to target specific genes that inhibit fetal hemoglobin production, allowing for a more effective treatment. Patients will have their stem cells edited and then reintroduced into their bodies to produce healthier red blood cells.
Why it matters
Potential benefit: If successful, this research could provide a potentially curative treatment for individuals suffering from sickle cell disease.
How similar studies have gone: Previous research using similar gene editing techniques has shown promising results in increasing fetal hemoglobin levels, indicating potential success for this approach.
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.