Base editing and prime editing for sickle cell disease
Funded at St. Jude Children's Research Hospital · NIH-11413777 · Data updated 2026-11-12
Aims to improve treatment for adults with sickle cell disease by using precise genome editing to boost fetal hemoglobin or correct the sickle mutation.
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 start | January 1, 2021 |
| Project end | June 30, 2030 |
| Funding year | 2026 |
| Award (this year) | $765,484 |
| NIH project number | 5R01HL156647-06 |
| Project ID | NIH-11413777 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults 21 years and older with sickle cell disease who may be candidates for autologous genome-edited cell therapies.
✕Less likely to help
Patients who cannot undergo autologous cell therapy or have conditions that preclude genome editing may not benefit.
What this research studies
This project focuses on new genome-editing approaches to treat sickle cell disease in adults. Researchers plan to use base editing to turn on fetal hemoglobin and prime editing to correct the disease-causing mutation in patient-derived cells, potentially enabling autologous therapies with fewer side effects. The work builds on recent approvals of genome-edited autologous therapies and seeks safer, more effective conditioning for engraftment. The study is conducted at St. Jude Children’s Research Hospital, led by Mitchell J. Weiss, and involves translating lab findings toward a clinical-ready approach for adults with SCD.
Why it matters
Potential benefit: If successful, this research could lead to safer, more effective gene-editing therapies that reduce pain, organ damage, and mortality in adults with sickle cell disease.
How similar studies have gone: Previous genome-editing efforts in SCD have shown promise, with ongoing work addressing safety and efficacy improvements.
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
Related research
Other NIH-funded projects on the same topics.
-
Digital twins of the spleen to help design better therapies for sickle cell disease
A computer-driven replica of the spleen is being developed to speed up creating safer and more effective treatments for people with sickle cell disease.
-
Accelerating hydroxyurea use for sickle cell disease in Nigeria
A pragmatic study in Nigeria aims to help doctors use hydroxyurea more consistently for people with sickle cell disease.
-
Immune and nerve signals behind acupuncture relief for sickle cell pain
This NIH grant explores how acupuncture helps reduce pain and opioid use for people with sickle cell disease.
-
Mindfulness-based Support for Pain and Sleep in Young People with Sickle Cell Disease
A digital mindfulness program is being tailored for adolescents and young adults with sickle cell disease to help with pain and sleep and is being tested for practicality and early effectiveness.
-
How diet affects white blood cells and sickle cell disease
A study looks at how nutrition, from early life to adulthood, influences sickle cell disease by examining neutrophils and disease severity in people with SCD and in a mouse model.
-
Nutrition-based plan to ease pain in sickle cell disease
A nutrition-based approach is being tested to reduce pain for people living with sickle cell disease.
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.