Gene therapy for brain diseases using cerebrospinal fluid delivery
Official title: Translational studies on cerebrospinal fluid (CSF)-directed gene therapy for global neurometabolic brain disease
Funded at Children's Hosp of Philadelphia · NIH-10599930 · Data updated 2026-11-12
This study is exploring a new way to treat alpha-mannosidosis, a genetic brain condition, by using gene therapy delivered directly into the fluid around the brain to help fix the underlying issues and improve brain function.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | April 1, 2019 |
| Project end | March 31, 2026 |
| Funding year | 2023 |
| Award (this year) | $612,884 |
| NIH project number | 5R01NS110349-05 |
| Project ID | NIH-10599930 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with alpha-mannosidosis or similar lysosomal storage disorders.
✕Less likely to help
Patients with brain diseases not related to lysosomal storage disorders may not benefit from this research.
What this research studies
This research investigates a novel approach to treat inherited brain diseases by delivering gene therapy directly into the cerebrospinal fluid (CSF). The focus is on a specific condition called alpha-mannosidosis, which affects brain function due to a genetic defect. By using a viral vector to introduce a normal gene into affected brain cells, the therapy aims to correct the metabolic issues throughout the brain. This method has the potential to improve treatment outcomes by ensuring widespread distribution of the therapeutic gene.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for patients with global neurometabolic brain diseases.
How similar studies have gone: Previous research has shown promise in using gene therapy for lysosomal storage disorders, indicating potential success for this approach.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Wolfe, John H — Children's Hosp of Philadelphia
- Study coordinator: Wolfe, John H
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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.