Introducing genes into the human genome without using donor DNA or causing DNA breaks
Human genetic supplementation without donor DNA or a DNA break
This study is exploring a new way to safely add genes to your DNA without causing any damage, which could help improve treatments for genetic disorders and make sure your body can produce the proteins it needs.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California Berkeley NIH-funded |
| Lab location | 1 site (Berkeley, United States) |
| Project ID | NIH-11165290 on NIH RePORTER |
What this research studies
This research investigates a novel method for gene therapy that aims to introduce genes into the human genome without the need for donor DNA or creating DNA breaks, which can be harmful. The approach focuses on overcoming the limitations of current CRISPR-based techniques, which often lead to non-specific integration and low efficiency. By developing a non-mutagenic and non-toxic method for gene introduction, this research seeks to provide a safer and more effective solution for treating various genetic disorders. Patients may benefit from this innovative technology, which could enable better control of protein expression and address loss-of-function pathologies.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic disorders that could benefit from gene therapy.
Not a fit: Patients with conditions that do not involve loss-of-function genetic issues may not receive benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective gene therapies for patients with genetic disorders.
How similar studies have performed: While gene therapy is a well-explored field, this specific approach is novel and has not been extensively tested in prior research.
Where this research is happening
Berkeley, United States
- University of California Berkeley — Berkeley, United States (Active)
Researchers
- Principal investigator: Collins, Kathleen — University of California Berkeley
- Study coordinator: Collins, Kathleen
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.