Creating a new treatment for a genetic neurological disorder using AAV vectors
MANUFACTURING OF PLASMID AND VECTOR AND GLP DOSE-ESCALATION TOXICOLOGY STUDIES
This study is working on a new treatment for Aspartylglucosaminuria, a serious genetic brain disorder, using a special delivery system to help make sure it’s safe and effective for patients who currently have limited options.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Battelle Centers/pub Hlth Res & Evaluatn NIH-funded |
| Lab location | 1 site (Columbus, United States) |
| Project ID | NIH-11172202 on NIH RePORTER |
What this research studies
This research focuses on developing an AAV9 vector and plasmids as a potential therapy for Aspartylglucosaminuria, a severe genetic neurological disorder. The project involves manufacturing the vector and conducting Good Laboratory Practice (GLP) dose-escalation toxicology studies to ensure safety and efficacy. Patients may benefit from this innovative approach as it aims to provide a new treatment option for a currently underserved condition. The research will include rigorous testing to confirm the quality and effectiveness of the manufactured products.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Aspartylglucosaminuria or related genetic neurological disorders.
Not a fit: Patients with other unrelated neurological disorders or those not diagnosed with Aspartylglucosaminuria may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a new therapeutic option for patients suffering from Aspartylglucosaminuria.
How similar studies have performed: Similar approaches using AAV vectors have shown promise in other genetic therapies, indicating potential for success in this novel application.
Where this research is happening
Columbus, United States
- Battelle Centers/pub Hlth Res & Evaluatn — Columbus, United States (Active)
Researchers
- Principal investigator: Burnaugh, Amanda — Battelle Centers/pub Hlth Res & Evaluatn
- Study coordinator: Burnaugh, Amanda
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.