Using CRISPR technology to improve gene editing for Sickle Cell Disease treatment
Official title: CRISPR-based transistors for high throughput multiplexed monitoring of CRISPR-based editing efficiency for Sickle cell disease
Funded at University of California, San Diego · NIH-11092291 · Data updated 2026-11-12
This study is working on a new way to use CRISPR technology to make gene editing better for people with Sickle Cell Disease, so they can have more effective treatments and improved health outcomes.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project start | January 19, 2024 |
| Project end | February 28, 2026 |
| Funding year | 2025 |
| Award (this year) | $388,206 |
| NIH project number | 5R01HL161361-04 |
| Project ID | NIH-11092291 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Sickle Cell Disease who may benefit from advanced gene therapy techniques.
✕Less likely to help
Patients with Sickle Cell Disease who are not eligible for gene therapy or those with other underlying health conditions may not receive benefit from this research.
What this research studies
This research focuses on developing a new platform that uses CRISPR technology to enhance the efficiency of gene editing for patients with Sickle Cell Disease (SCD). By creating multiplex CRISPR-transistors, the team aims to monitor and optimize how effectively gene editing occurs in blood stem cells. This approach seeks to address the challenges of low editing efficiency that can hinder the effectiveness of potential gene therapies. Patients may benefit from improved treatment outcomes through more effective gene editing techniques.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies for Sickle Cell Disease, potentially improving patient outcomes and quality of life.
How similar studies have gone: Previous research has shown promise in using CRISPR technology for gene editing, indicating that this approach could lead to significant advancements in treatment.
Where this research is happening
-
University of California, San Diego
La Jolla, United States Active
Researchers
- Principal investigator: Aran, Kiana — University of California, San Diego
- Study coordinator: Aran, Kiana
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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.