New precise gene-editing tools to fix single-letter DNA errors
Engineering Novel Precision Genome Editing Tools
This project is creating more accurate CRISPR-based editors to correct single-letter DNA changes that cause inherited conditions like some neurologic disorders and cystic fibrosis.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California-Irvine NIH-funded |
| Lab location | 1 site (Irvine, United States) |
| Project ID | NIH-11360437 on NIH RePORTER |
What this research studies
Researchers will use molecular insights about current base editors to design four new strategies that cut down unwanted edits, improve editing efficiency, and expand the types of single-letter changes that can be fixed. The work will involve testing redesigned editors in cells and laboratory models to measure accuracy, off-target effects, and the ability to correct previously untreatable mutations. The team aims to reduce bystander edits and allow correction of transversion mutations (changing a purine to a pyrimidine or vice versa). If successful, these lab advances would be stepping stones toward therapies that could one day be tested in people.
Who could benefit from this research
Good fit: Ideal candidates for future therapies enabled by this work are people whose disease is caused by a specific single-nucleotide mutation (SNP), such as some forms of cystic fibrosis or certain inherited neurologic disorders.
Not a fit: Patients whose conditions are not driven by single-letter DNA changes (for example polygenic or non-genetic diseases) or who need immediate clinical care are unlikely to benefit from this early-stage lab research.
Why it matters
Potential benefit: If successful, this could allow safer and more precise correction of disease-causing single-letter DNA mutations and broaden which genetic conditions can be treated.
How similar studies have performed: Related CRISPR base editors and prime editors have fixed mutations in cells and animal models but still show off-target edits and limited targeting scope, so this proposal builds on promising but imperfect prior work.
Where this research is happening
Irvine, United States
- University of California-Irvine — Irvine, United States (Active)
Researchers
- Principal investigator: Lapinaite, Audrone — University of California-Irvine
- Study coordinator: Lapinaite, Audrone
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.