Investigating how changes in cell structures affect brain development
Examining the Impact of Peroxisomal Fission on Cell Fate Decisions During Neurodevelopment
['FUNDING_FELLOWSHIP'] · VANDERBILT UNIVERSITY · NIH-10997487
This study is looking at Zellweger spectrum disorders, which are rare genetic conditions that affect brain development, and it aims to learn how changes in a specific gene can impact brain cell growth, with the hope of finding new treatments for those who have these conditions.
Quick facts
| Phase | ['FUNDING_FELLOWSHIP'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | VANDERBILT UNIVERSITY (nih funded) |
| Locations | 1 site (Nashville, UNITED STATES) |
| Trial ID | NIH-10997487 on ClinicalTrials.gov |
What this research studies
This research focuses on Zellweger spectrum disorders, which are rare genetic conditions that severely impact brain development and function. By using advanced techniques like CRISPR to manipulate a specific gene (PEX11b), the study aims to understand how changes in peroxisomal structures influence neurodevelopment. Researchers will analyze the effects of these changes on brain cell growth and function using human stem cells. The goal is to uncover mechanisms that could lead to new treatments for affected individuals.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Zellweger spectrum disorders or those who are carriers of mutations affecting peroxisomal biogenesis.
Not a fit: Patients with neurological disorders not related to peroxisomal dysfunction may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic strategies for treating severe neurological disorders associated with Zellweger spectrum disorders.
How similar studies have performed: Previous research has shown promising results in manipulating genetic factors related to neurodevelopment, suggesting potential for success in this novel approach.
Where this research is happening
Nashville, UNITED STATES
- VANDERBILT UNIVERSITY — Nashville, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BODNYA, CAROLINE — VANDERBILT UNIVERSITY
- Study coordinator: BODNYA, CAROLINE
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.