Introducing genes into the human genome without using donor DNA or causing DNA breaks
Human genetic supplementation without donor DNA or a DNA break
['FUNDING_OTHER'] · UNIVERSITY OF CALIFORNIA BERKELEY · NIH-10912717
This study is exploring a new and safer way to use gene therapy to help people with genetic disorders by introducing genes into their DNA without causing any damage, making it a friendlier option for treatment.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CALIFORNIA BERKELEY (nih funded) |
| Locations | 1 site (BERKELEY, UNITED STATES) |
| Trial ID | NIH-10912717 on ClinicalTrials.gov |
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 the creation of DNA breaks, which can be harmful. The approach seeks to overcome the limitations of current CRISPR-based techniques that often result in non-specific integration and low efficiency. By developing a non-mutagenic and non-toxic method, the research hopes to provide a safer and more effective way to treat genetic disorders caused by loss-of-function mutations. Patients may benefit from this innovative technology that could enhance gene therapy outcomes.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic disorders that could benefit from gene therapy, particularly those with loss-of-function mutations.
Not a fit: Patients with conditions that do not involve genetic mutations or those who are not candidates for gene therapy may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective gene therapies for various genetic disorders.
How similar studies have performed: While gene therapy is a well-explored field, this specific approach of non-mutagenic gene introduction without donor DNA is novel and has not been widely tested.
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.