Creating new technologies for precise gene editing in living cells
Development of Technologies for Efficient In Vivo Prime Editing
This study is working on a new way to change genes more safely and accurately, which could lead to better treatments for conditions like Amyotrophic Lateral Sclerosis, helping patients by targeting the root causes of their illness.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Illinois at Urbana-Champaign NIH-funded |
| Lab location | 1 site (Champaign, United States) |
| Project ID | NIH-10804609 on NIH RePORTER |
What this research studies
This research focuses on developing advanced gene editing technologies, specifically prime editing, which allows for precise modifications of DNA without causing double strand breaks. The approach involves creating a split-PE platform that can be delivered into living organisms using adeno-associated viruses (AAV), which are effective gene delivery vehicles. By overcoming current limitations in gene editing, this research aims to enhance the safety and efficacy of genetic therapies for conditions like Amyotrophic Lateral Sclerosis. Patients may benefit from improved treatment options that target the underlying genetic causes of their conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Amyotrophic Lateral Sclerosis or related genetic conditions.
Not a fit: Patients with non-genetic forms of motor neuron diseases may not receive any benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective and safer gene therapies for patients with genetic disorders.
How similar studies have performed: Other research has shown promise with similar gene editing technologies, indicating potential for success in this novel approach.
Where this research is happening
Champaign, United States
- University of Illinois at Urbana-Champaign — Champaign, United States (Active)
Researchers
- Principal investigator: Perez-Pinera, Pablo — University of Illinois at Urbana-Champaign
- Study coordinator: Perez-Pinera, Pablo
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.