Testing a new gene therapy for a severe childhood brain disease
Pre-clinical testing of a novel gene therapy for pediatric neurodegenerative disease
['FUNDING_R21'] · UT SOUTHWESTERN MEDICAL CENTER · NIH-11057384
This study is testing a new gene therapy for children with neurodegenerative diseases caused by genetic changes, aiming to fix the problem at its source by delivering a healthy version of a specific gene to help improve their brain and muscle function.
Quick facts
| Phase | ['FUNDING_R21'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UT SOUTHWESTERN MEDICAL CENTER (nih funded) |
| Locations | 1 site (DALLAS, UNITED STATES) |
| Trial ID | NIH-11057384 on ClinicalTrials.gov |
What this research studies
This research focuses on developing a gene therapy to treat pediatric neurodegenerative diseases caused by genetic mutations. It aims to deliver a functional copy of the VAC14 gene using an AAV9 virus, which can help restore normal cell function and potentially halt disease progression. The therapy is designed to address the underlying genetic issues rather than just managing symptoms, offering a more effective long-term solution for affected children. Patients will be monitored to assess the therapy's impact on their neurological and muscular health.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with childhood-onset striatonigral degeneration or similar neurodegenerative conditions caused by recessive gene mutations.
Not a fit: Patients with neurodegenerative diseases not caused by genetic mutations or those who are older than the targeted age group may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option that may halt or prevent the progression of severe pediatric neurodegenerative diseases.
How similar studies have performed: Recent advancements in gene therapy, such as the FDA approval for spinal muscular atrophy, suggest that similar approaches have shown success and could be promising for this research.
Where this research is happening
DALLAS, UNITED STATES
- UT SOUTHWESTERN MEDICAL CENTER — DALLAS, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: RIOS, JONATHAN — UT SOUTHWESTERN MEDICAL CENTER
- Study coordinator: RIOS, JONATHAN
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.
Conditions: Aran-Duchenne disease