Investigating how mitochondrial mRNA structure affects Leigh Syndrome
Mitochondrial mRNA structure as a driver of Leigh Syndrome
This study is looking at how certain changes in proteins can affect the way genes work in children with Leigh syndrome, a serious condition that causes developmental delays, to help find new ways to treat the disease.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Miami School of Medicine NIH-funded |
| Lab location | 1 site (Coral Gables, United States) |
| Project ID | NIH-11005001 on NIH RePORTER |
What this research studies
This research focuses on Leigh syndrome, a severe mitochondrial disease that leads to developmental regression in children. The team will explore how mutations in specific proteins, LRPPRC and TACO1, disrupt the structure of mitochondrial mRNAs, which may contribute to the disease's progression. By utilizing advanced RNA structure profiling techniques, the researchers aim to uncover the molecular mechanisms behind these mutations and their impact on gene expression. This could provide insights into potential therapeutic targets for treating Leigh syndrome.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with Leigh syndrome, particularly those under the age of two.
Not a fit: Patients with Leigh syndrome who are older than two years or those without a confirmed diagnosis may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new treatment options for children affected by Leigh syndrome.
How similar studies have performed: While research on mitochondrial diseases is ongoing, this specific approach using RNA structure profiling is relatively novel and has not been extensively tested in the context of Leigh syndrome.
Where this research is happening
Coral Gables, United States
- University of Miami School of Medicine — Coral Gables, United States (Active)
Researchers
- Principal investigator: Moran, John Conor — University of Miami School of Medicine
- Study coordinator: Moran, John Conor
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.