Gene Therapy for Alpha-Mannosidosis in the Brain
Gene Transfer and NMR Studies in Alpha-Mannosidosis Brain
This research explores a new way to deliver a healthy gene to the brain to help treat alpha-mannosidosis, a genetic condition affecting many brain cells.
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 ID | NIH-11159858 on NIH RePORTER |
What this research studies
For conditions like alpha-mannosidosis, where a genetic problem affects many brain cells, it's challenging to deliver treatment widely. This project is developing a method to introduce a normal gene into some brain cells, which then produce a therapeutic enzyme that can spread to neighboring cells. The goal is to achieve widespread correction throughout the brain, overcoming limitations seen with current delivery methods in larger animals. Researchers are testing a new gene delivery system using a special virus to ensure the treatment reaches all affected areas.
Who could benefit from this research
Good fit: Future patients with alpha-mannosidosis, particularly those with severe forms affecting the brain, could potentially benefit from the therapies developed through this research.
Not a fit: Patients without alpha-mannosidosis or those whose condition does not involve the specific genetic defect targeted by this gene therapy may not directly benefit.
Why it matters
Potential benefit: If successful, this work could lead to a new gene therapy that effectively treats the widespread brain problems in patients with alpha-mannosidosis.
How similar studies have performed: Previous work in this project has shown promising results in an animal model, demonstrating widespread gene delivery and correction of disease markers using a novel delivery method.
Where this research is happening
Philadelphia, United States
- 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
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.