Targeting RNA to control gene expression
Modulating gene expression by RNA-targeting chimeras
This study is exploring new ways to create special molecules that can attach to RNA, which might help change how genes work in people with genetic disorders and diseases that current treatments can't effectively target.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Chicago NIH-funded |
| Lab location | 1 site (Chicago, United States) |
| Project ID | NIH-11084498 on NIH RePORTER |
What this research studies
This research focuses on developing innovative molecules that can specifically bind to RNA, allowing for the modulation of gene expression in conditions where traditional drug targets are ineffective. By creating chimeric molecules inspired by existing technologies, the team aims to enhance the specificity and predictability of RNA-targeting agents. Patients may benefit from these advancements as they could lead to new treatments for genetic disorders and diseases previously deemed 'undruggable'.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic disorders that involve undruggable gene products, such as those affected by Aran-Duchenne disease.
Not a fit: Patients with conditions that do not involve RNA-targeted therapies or those whose genetic issues are not related to the specific genes being studied may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide new therapeutic options for patients with genetic disorders by enabling targeted modulation of gene expression.
How similar studies have performed: Previous research has shown success with RNA-targeting approaches, such as the approval of Risdiplam for spinal muscular atrophy, indicating potential for this novel methodology.
Where this research is happening
Chicago, United States
- University of Chicago — Chicago, United States (Active)
Researchers
- Principal investigator: Wang, Jingxin — University of Chicago
- Study coordinator: Wang, Jingxin
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.