Improving gene editing techniques for better treatment of diseases
Towards Robust Multiplex Genome Engineering Beyond CRISPR-Cas9
This study is working on improving gene-editing tools to make them more precise and effective for treating diseases like cancer and autoimmune conditions, so that doctors can better help patients with these health issues.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project ID | NIH-10994585 on NIH RePORTER |
What this research studies
This research focuses on enhancing gene-editing technologies beyond the current CRISPR-Cas9 system to address its limitations, such as low efficiency and off-target effects. The approach involves designing new microbial proteins that can perform precise genome modifications and developing advanced methods for tracking genetic changes at the single-cell level. By validating these tools in human cancer models, the research aims to provide more accurate and effective gene-editing solutions for various diseases, including autoimmune conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic disorders or autoimmune diseases that could benefit from advanced gene-editing therapies.
Not a fit: Patients with conditions that do not involve genetic mutations or require gene editing may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more precise and effective treatments for genetic diseases and cancers.
How similar studies have performed: Other research has shown promise in improving gene-editing techniques, but this approach aims to address specific limitations of existing methods, making it a novel endeavor.
Where this research is happening
Stanford, United States
- Stanford University — Stanford, United States (Active)
Researchers
- Principal investigator: Cong, Le — Stanford University
- Study coordinator: Cong, Le
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.