Gene-editing to create better mouse models for complex diseases like Alzheimer's
Development of approaches to apply CRISPR/Cas9-mediated gene conversion to model complex genetic traits in mice
Researchers are using CRISPR gene-editing to make mice carry combinations of Alzheimer's-linked genes so future treatments can be developed and tested more reliably.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-11326330 on NIH RePORTER |
What this research studies
From a patient's perspective, scientists will use CRISPR/Cas9 in mouse germ cells to copy specific disease-linked gene variants from one chromosome to another so that offspring inherit the desired combinations of genes. The team will design guide RNAs and limit Cas9 activity to the germline, then breed and genotype animals to confirm gene conversion events. Resulting mice with coordinated human-relevant gene combinations will be characterized for Alzheimer's-like biology to see if they better reflect the human condition. This approach aims to reduce the number of animals, time, and cost needed to model complex genetic risk.
Who could benefit from this research
Good fit: This is a lab-based project using mice and does not enroll patients, so there are no patient participants to join.
Not a fit: People looking for immediate new treatments for Alzheimer's are unlikely to benefit directly, as this work is preclinical and intended to improve future research tools.
Why it matters
Potential benefit: If successful, this could speed the creation of more accurate animal models of Alzheimer's, which may help researchers find effective treatments faster.
How similar studies have performed: CRISPR-based gene editing has worked in mice in early preclinical settings, but using germline gene conversion to model complex multi-gene human traits is relatively new and experimental.
Where this research is happening
La Jolla, United States
- University of California, San Diego — La Jolla, United States (Active)
Researchers
- Principal investigator: Cooper, Kimberly Lynn — University of California, San Diego
- Study coordinator: Cooper, Kimberly Lynn
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.