A new method for delivering gene editing to treat neurogenetic diseases.
Official title: A non-viral CRISPR-mediated genome editing delivery platform as a potential therapy for neurogenetic diseases
Funded at Yale University · NIH-10932377 · Data updated 2026-11-12
This study is testing a new way to deliver gene editing treatments directly into the spinal fluid to help people with neurogenetic diseases like Angelman syndrome and H1-4 syndrome, aiming to fix a key gene that isn't working properly.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Yale University NIH-funded |
| Lab location | 1 site (New Haven, United States) |
| Project start | September 20, 2023 |
| Project end | July 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $10,834,632 |
| NIH project number | 5UG3TR004713-02 |
| Project ID | NIH-10932377 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include children and adults diagnosed with Angelman syndrome or H1-4 syndrome.
✕Less likely to help
Patients with neurogenetic disorders not related to Angelman syndrome or H1-4 syndrome may not benefit from this research.
What this research studies
This research focuses on developing a safe and effective delivery system for CRISPR-based gene editing specifically targeting neurogenetic diseases like Angelman syndrome and H1-4 syndrome. The approach utilizes a chemically modified ribonucleoprotein (cRNP) that can be administered directly into the spinal fluid, allowing it to reach neuronal cells in the brain. By reducing the expression of a specific antisense RNA, the therapy aims to restore the function of a critical gene involved in these disorders. This innovative method seeks to overcome the challenges of traditional gene therapy delivery.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients with severe neurogenetic disorders, potentially improving their neurological function and quality of life.
How similar studies have gone: Previous research has shown promise in using CRISPR and antisense oligonucleotides for similar genetic conditions, indicating a potential for success with this novel delivery approach.
Where this research is happening
-
Yale University
New Haven, United States Active
Researchers
- Principal investigator: Jiang, Yong-Hui — Yale University
- Study coordinator: Jiang, Yong-Hui
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.