A new method for editing genes and their regulation in mice
A modular system for murine CRISPR genome and epigenome editing
This study is working on new ways to change genes in mice using a special technology called CRISPR, which could help scientists better understand how genes work and how they relate to diseases.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11145318 on NIH RePORTER |
What this research studies
This research focuses on developing advanced tools for editing genes and their regulatory elements in mice using CRISPR technology. By creating mouse models that express specific CRISPR components, the researchers aim to enhance the precision and efficiency of genetic modifications. The approach involves using adeno-associated virus (AAV) vectors to deliver these editing tools, allowing for more effective and uniform gene editing compared to traditional methods. This innovative strategy could lead to significant advancements in understanding genetic functions and diseases.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic conditions that could benefit from advanced gene editing technologies.
Not a fit: Patients with conditions that are not related to genetic mutations or do not require gene editing may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies and treatments for genetic disorders.
How similar studies have performed: Previous research has shown promising results using CRISPR technology for gene editing, indicating that this approach has the potential for success.
Where this research is happening
Boston, United States
- Boston Children's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Pu, William Tswenching — Boston Children's Hospital
- Study coordinator: Pu, William Tswenching
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.