Improving Gene Therapy for Muscular Dystrophy
Project 2 - McNally (NU)_Spencer (UCLA)
['FUNDING_OTHER'] · UNIVERSITY OF FLORIDA · NIH-11130233
This research aims to make gene therapy safer and more effective for people living with muscular dystrophies that affect both muscles and the heart.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF FLORIDA (nih funded) |
| Locations | 1 site (GAINESVILLE, UNITED STATES) |
| Trial ID | NIH-11130233 on ClinicalTrials.gov |
What this research studies
We are working to improve gene therapy, a treatment that delivers healthy genes to replace faulty ones, for conditions like Limb Girdle Muscular Dystrophy (LGMD) and Duchenne Muscular Dystrophy (DMD). These conditions often weaken both skeletal muscles and the heart, so our goal is to find ways to correct issues in both areas. We are particularly focused on understanding how the body's immune system reacts to the gene therapy delivery method, called AAV vectors, to make future treatments more successful and reduce side effects. Our work uses advanced models to test new strategies before they reach patients.
Who could benefit from this research
Good fit: This research is relevant for patients with Limb Girdle Muscular Dystrophy (LGMD), particularly those with β-sarcoglycan- and FKRP-related types, and Duchenne Muscular Dystrophy (DMD).
Not a fit: Patients without these specific types of muscular dystrophy or other conditions not targeted by gene therapy for sarcoglycan or dystrophin may not directly benefit from this particular research.
Why it matters
Potential benefit: If successful, this work could lead to safer and more effective gene therapy treatments for patients with muscular dystrophies, improving both muscle strength and heart function.
How similar studies have performed: There has been progress in human gene therapy for muscular dystrophy, and human AAV clinical trials have already begun for some LGMD subtypes, indicating a foundation of prior work.
Where this research is happening
GAINESVILLE, UNITED STATES
- UNIVERSITY OF FLORIDA — GAINESVILLE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: MCNALLY, ELIZABETH M — UNIVERSITY OF FLORIDA
- Study coordinator: MCNALLY, ELIZABETH M
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.