A new method for delivering gene editing to treat neurogenetic diseases.
A non-viral CRISPR-mediated genome editing delivery platform as a potential therapy for neurogenetic diseases
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 ID | NIH-10932377 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research include children and adults diagnosed with Angelman syndrome or H1-4 syndrome.
Not a fit: Patients with neurogenetic disorders not related to Angelman syndrome or H1-4 syndrome may not benefit from this research.
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 performed: 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
New Haven, United States
- 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 below.