Non-viral CRISPR delivery to treat Angelman syndrome and related neurodevelopmental disorders
Official title: A non-viral CRISPR-mediated genome editing delivery platform as a potential therapy for neurogenetic diseases
Funded at Yale University · NIH-11582654 · Data updated 2026-11-12
A new, non-viral gene editing delivery system is being developed to target brain cells and potentially treat Angelman syndrome and related neurodevelopmental disorders.
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, 2027 |
| Funding year | 2026 |
| Award (this year) | $3,500,001 |
| NIH project number | 3UG3TR004713-03S1 |
| Project ID | NIH-11582654 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with Angelman syndrome or HIST1H1E syndrome who could be eligible for future gene-editing therapies and have access to specialized neurogenetic care.
✕Less likely to help
Patients without these specific neurogenetic conditions or those seeking immediate treatments may not benefit from this early-stage platform.
What this research studies
This grant supports advancing a chemically modified ribonucleoprotein delivery platform that reaches neuronal cells through intrathecal administration. The goal is to apply this technology to Angelman syndrome and HIST1H1E syndrome, aiming to restore proper gene expression in the brain. The work involves early-stage development and testing in models to refine safety, specificity, and effectiveness before any patient trials. If successful, the approach could offer a one-size-fits-many delivery tool for neurogenetic diseases using patient-relevant biology.
Why it matters
Potential benefit: If successful, patients with Angelman syndrome or HIST1H1E syndrome could eventually benefit from a safer, more targeted gene editing therapy that addresses root genetic causes.
How similar studies have gone: Previous work has shown promise in gene editing approaches to correct disease mechanisms in models, supporting the potential for translational impact but this platform is still in early development.
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.