Creating advanced tools for precise genome editing
Engineering Novel Precision Genome Editing Tools
['FUNDING_OTHER'] · UNIVERSITY OF CALIFORNIA-IRVINE · NIH-11136146
This study is working on new tools to fix tiny genetic changes that can cause diseases, aiming to make treatments safer and more effective for people with genetic disorders.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CALIFORNIA-IRVINE (nih funded) |
| Locations | 1 site (IRVINE, UNITED STATES) |
| Trial ID | NIH-11136146 on ClinicalTrials.gov |
What this research studies
This research focuses on developing innovative genome editing tools that can accurately correct genetic variations known as single nucleotide polymorphisms (SNPs), which are linked to various diseases. The approach aims to enhance the efficiency and precision of current CRISPR-Cas based technologies, addressing their limitations such as off-target effects and low editing efficiency. By exploring new strategies, the research seeks to create tools that can perform targeted edits without causing unintended changes to the genome, potentially leading to safer and more effective therapies for genetic disorders.
Who could benefit from this research
Good fit: Ideal candidates for this research would include individuals with genetic disorders caused by specific SNPs that could be targeted for correction.
Not a fit: Patients with genetic conditions not caused by SNPs or those whose conditions are not amenable to genome editing may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for genetic diseases by enabling precise corrections of harmful genetic variations.
How similar studies have performed: While genome editing technologies like CRISPR have shown promise, this research aims to address specific limitations and expand the capabilities of these tools, making it a novel approach in the field.
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.