Targeted delivery systems to edit blood stem cells
Official title: Engineering Targeted Delivery Vehicles for Genome Editing in Hematopoietic Stem Cells
Funded at J. David Gladstone Institutes · NIH-11439543 · Data updated 2026-11-12
Aims to safely deliver genome-editing tools to blood stem cells to treat diseases like acute myeloid leukemia.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | J. David Gladstone Institutes NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | July 10, 2026 |
| Project end | June 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $485,966 |
| NIH project number | 1R21CA318067-01 |
| Project ID | NIH-11439543 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with conditions involving blood or immune system disorders where future stem-cell–targeted gene editing could be beneficial.
✕Less likely to help
If you do not have a blood or immune system disorder that could be addressed by stem-cell editing, direct benefits are unlikely in the near term.
What this research studies
This project explores engineered delivery vehicles that carry CRISPR–Cas9 components directly to hematopoietic stem cells. Researchers will adjust antibody fragments on the delivery surface to control how well these cells take up the editing tools and how precisely editing occurs. The work builds on previous EDV technology to minimize off-target effects and maximize genome editing in the right cell type. By focusing on hematopoietic stem cells, the study targets a key point in blood cancers and related disorders. The timeline spans two years, with experiments designed to map how binding strength and density influence delivery efficiency.
Why it matters
Potential benefit: If successful, this approach could enable precise genetic modification of blood stem cells to treat inherited or acquired blood disorders, including certain leukemias.
How similar studies have gone: Previous work with envelope-delivery systems and CRISPR components shows potential for targeted editing, though clinical translation remains ongoing.
Where this research is happening
-
J. David Gladstone Institutes
San Francisco, United States Active
Researchers
- Principal investigator: Doudna, Jennifer a — J. David Gladstone Institutes
- Study coordinator: Doudna, Jennifer a
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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.