Precise gene fixes for sickle cell disease using base and prime editing
Base editing and prime editing for sickle cell disease
['FUNDING_R01'] · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · NIH-11228251
This project uses two newer gene‑editing approaches—base editing to raise protective fetal hemoglobin and prime editing to correct the sickle mutation—in adults with sickle cell disease.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | ST. JUDE CHILDREN'S RESEARCH HOSPITAL (nih funded) |
| Locations | 1 site (MEMPHIS, UNITED STATES) |
| Trial ID | NIH-11228251 on ClinicalTrials.gov |
What this research studies
If you join, your own blood stem cells would be collected and edited in the lab using either base editing to increase fetal hemoglobin or prime editing to directly fix the sickle mutation. The edited cells would then be returned to you after a reduced‑toxicity bone marrow conditioning regimen designed to help them engraft. The team aims to make more precise DNA changes with fewer double‑strand breaks than older Cas9 or viral methods, potentially lowering long‑term risk. The work builds on autologous stem‑cell gene therapies but tests next‑generation editing techniques and safer conditioning strategies.
Who could benefit from this research
Good fit: Adults (age 21 and older) with sickle cell disease who are medically fit for autologous hematopoietic stem‑cell collection and conditioning would be the ideal candidates.
Not a fit: People who are too frail or have medical conditions that prevent stem‑cell procedures, children below the study age, or those ineligible for bone marrow conditioning are unlikely to be eligible or benefit.
Why it matters
Potential benefit: If successful, this could offer a more precise, potentially safer one‑time genetic treatment or cure for sickle cell disease with less toxic conditioning.
How similar studies have performed: Very recently, FDA‑approved HSC gene therapies using Cas9 or lentiviral vectors have shown clinical benefit, while base and prime editing are more precise approaches that have less human clinical experience so far.
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.