Improving gene editing in blood stem cells using nanoparticles
Official title: R15: Enhancement of Genome Editing in Hematopoietic Stem Cells by Nucleic Acids Nanoparticles Delivery of CRISPR/CAS System
Funded at Rutgers the State Univ of Nj Camden · NIH-11043864 · Data updated 2026-11-12
This study is looking at a new way to help deliver a gene-editing tool called CRISPR to blood stem cells, which could lead to better treatments for genetic blood disorders like sickle cell disease.
Quick facts
| Grant type | R15 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Rutgers the State Univ of Nj Camden NIH-funded |
| Lab location | 1 site (Camden, United States) |
| Project start | February 1, 2025 |
| Project end | January 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $486,291 |
| NIH project number | 1R15HL177745-01 |
| Project ID | NIH-11043864 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic blood disorders, such as sickle cell disease, who may benefit from advanced gene editing therapies.
✕Less likely to help
Patients with non-genetic blood disorders or those who do not have hematopoietic stem cell-related conditions may not receive benefits from this research.
What this research studies
This research focuses on enhancing the delivery of the CRISPR/Cas9 gene editing system into hematopoietic stem cells (HSCs) using innovative nucleic acids nanoparticles (NANPs). By optimizing the design of these nanoparticles, the research aims to improve the efficiency and precision of gene editing, which is crucial for treating genetic blood disorders like sickle cell disease. The project will involve multidisciplinary approaches to evaluate how well these nanoparticles can deliver the CRISPR/Cas9 system and the effectiveness of the resulting gene modifications. If successful, this could lead to safer and more effective treatments for patients with blood-related genetic conditions.
Why it matters
Potential benefit: If successful, this research could provide more effective and safer gene therapies for patients with genetic blood disorders.
How similar studies have gone: Other research has shown promise in using nanoparticle technology for gene delivery, indicating potential success for this innovative approach.
Where this research is happening
-
Rutgers the State Univ of Nj Camden
Camden, United States Active
Researchers
- Principal investigator: Fu, Jinglin — Rutgers the State Univ of Nj Camden
- Study coordinator: Fu, Jinglin
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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.