AAV gene therapy to treat MPS IIIC neuropathy
Official title: Novel AAV gene therapy for treating CNS neuropathy of MPS IIIC
Funded at Neurogt, INC. · NIH-11369132 · Data updated 2026-11-12
A specialized gene therapy using an AAV9 vector is being developed to address the brain and nerve damage in people with MPS IIIC.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Neurogt, INC. NIH-funded |
| Lab location | 1 site (Chapel Hill, UNITED STATES) |
| Project start | July 8, 2025 |
| Project end | April 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $342,740 |
| NIH project number | 5R41NS143578-02 |
| Project ID | NIH-11369132 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with MPS IIIC or related lysosomal storage disorders who may benefit from a system-wide gene therapy approach in the future.
✕Less likely to help
Patients with conditions not driven by HGSNAT-related pathology or those unable to receive systemic AAV9 delivery may not benefit.
What this research studies
This project aims to create an AAV9-based gene therapy to fix the root cause of MPS IIIC, a serious lysosomal storage disease. It focuses on delivering the gene product to as many nervous system cells as possible, addressing global neuropathology. The researchers are exploring how extracellular vesicles might help spread the therapy between cells, potentially allowing lower doses and reducing risks. The work is in early development, considering delivery challenges and safety as the therapy is scaled toward human use.
Why it matters
Potential benefit: If successful, this approach could slow or halt nerve damage in MPS IIIC and improve neurological outcomes.
How similar studies have gone: Gene therapy approaches for MPS-related disorders have shown promise in preclinical models, though translating to humans remains challenging.
Where this research is happening
-
Neurogt, INC.
Chapel Hill, United States Active
Researchers
- Principal investigator: Fu, Haiyan — Neurogt, INC.
- Study coordinator: Fu, Haiyan
Related research
Other NIH-funded projects on the same topics.
-
Non-invasive, microscopic biomarkers to detect CIPN in chemotherapy patients
This grant aims to develop a non-invasive imaging method to measure nerve changes in patients undergoing chemotherapy to better understand and monitor chemotherapy-induced peripheral neuropathy (CIPN).
-
Neonatal Fc receptor blockade by efgartigimod to help inflammatory nerve disease
Aims to test how blocking a specific antibody trafficking receptor with efgartigimod could improve nerve inflammation in a model of AIDP/GBS-type neuropathy.
-
Schwann cell aging and secretions linked to diabetic nerve damage
A research project looking at how aging nerve cells and their secretions may drive nerve damage in people with type 2 diabetes.
-
Diabetic bladder problems in diabetes mice show sex-specific nerve and inflammation roles
Aims to understand why bladder problems happen in diabetes, exploring how inflammation and nerves differ between male and female mice.
-
Prenyltransferase inhibitor as a new therapy for inflammatory neuropathy
A new drug candidate called FGTI-2734 is being studied to calm nerve inflammation in conditions like AIDP/Guillain-Barré syndrome, with the goal of protecting nerves and reducing symptoms.
-
KCTD11-related CMT neuropathy: uncovering how it damages nerves
We are studying a genetic form of Charcot–Marie–Tooth disease to understand what goes wrong in nerves and support future treatments.
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.