New genetic techniques to treat sickle cell disease
Base editing and prime editing for sickle cell disease
This study is looking at new ways to use genetic editing to help people with sickle cell disease by safely fixing the genetic issues in their own blood cells, aiming to make treatment easier and more effective than current methods.
Quick facts
| Grant type | R01 grant |
|---|---|
| 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-10795850 on NIH RePORTER |
What this research studies
This research focuses on developing innovative genetic editing methods to treat sickle cell disease (SCD), a condition that causes severe pain and organ damage. The team will explore two advanced techniques, adenosine base editing and prime editing, which aim to correct the genetic mutations responsible for SCD without causing harmful DNA breaks. By modifying a patient's own blood stem cells, the goal is to provide a safer and more effective treatment option compared to traditional stem cell transplants. The research will evaluate the effectiveness of these techniques in creating beneficial genetic changes that could alleviate the symptoms of SCD.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults aged 21 and older who have been diagnosed with sickle cell disease.
Not a fit: Patients who do not have sickle cell disease or those who are not eligible for genetic modification therapies may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a safer and more accessible cure for sickle cell disease, improving patients' quality of life.
How similar studies have performed: While genetic editing for sickle cell disease is a relatively new approach, preliminary studies have shown promise in using similar techniques for other genetic disorders.
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.