Creating better tools to activate genes using CRISPR technology
Development of potent and predictable Cas9 gene activation tools through high-throughput screening
This study is working on making a better tool to help turn on genes in cells, which could lead to improved treatments for genetic disorders that might help patients in the future.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brigham and Women's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-10896340 on NIH RePORTER |
What this research studies
This research focuses on improving the CRISPR-Cas9 technology to enhance the activation of genes in cells. By using high-throughput screening methods, the team aims to develop more effective and reliable tools that can increase gene expression in both mouse and human cells. The approach involves testing various gene-activating peptides and their combinations to find the most potent options. Patients may benefit from advancements in gene therapy and treatments for genetic disorders as a result of this work.
Who could benefit from this research
Good fit: Ideal candidates for participation or benefit from this research would include individuals with genetic disorders that could be treated through gene activation.
Not a fit: Patients with conditions that are not related to genetic expression or those who do not have a genetic basis for their condition may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies for a variety of genetic conditions.
How similar studies have performed: Other research has shown promise in using CRISPR technology for gene activation, indicating that this approach has potential for success.
Where this research is happening
Boston, United States
- Brigham and Women's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Sherwood, Richard I — Brigham and Women's Hospital
- Study coordinator: Sherwood, Richard I
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.