Non-viral CRISPR delivery to treat Angelman and related neurogenetic disorders
A non-viral CRISPR-mediated genome editing delivery platform as a potential therapy for neurogenetic diseases
This project develops a non-viral CRISPR method to correct the gene problem that causes Angelman syndrome and a related neurodevelopmental disorder.
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-11195559 on NIH RePORTER |
What this research studies
This work is building a chemically modified CRISPR ribonucleoprotein (cRNP) delivery system that can be given into the spinal fluid (intrathecal) to reach neurons across the brain. The team plans to use that system to turn back on the UBE3A gene in Angelman syndrome by reducing the antisense RNA that silences it, and to address the mutant protein effect in HIST1H1E (H1-4) syndrome. Experiments will test delivery, distribution in the brain, and whether the approach restores normal gene function and improves disease-related features in preclinical models. The broader aim is to create a safer, quicker-to-develop delivery platform that could be used for multiple neurogenetic conditions.
Who could benefit from this research
Good fit: People with genetically confirmed Angelman syndrome or HIST1H1E-related neurodevelopmental disorder, including children and adults, would be the primary candidates for this line of work and for future trials.
Not a fit: Patients without these specific genetic diagnoses or whose symptoms are caused by different genes are unlikely to benefit from this approach.
Why it matters
Potential benefit: If successful, this approach could restore missing or abnormal brain proteins and offer a one-time or long-lasting genetic therapy that improves neurological symptoms in affected patients.
How similar studies have performed: Other methods like antisense oligonucleotides, topoisomerase inhibitors, and viral Cas9 have reactivated UBE3A and helped mouse models, but non-viral intrathecal CRISPR delivery to the brain is newer and less tested.
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.