Ultrasound-activated gene editing in the brain using light-controlled CRISPR-CMINI
Official title: Ultrasound-Controlled in Vivo Gene Editing with Photoswitchable CRISPR-CasMINI
Funded at Stanford University · NIH-11451108 · Data updated 2026-11-12
A Stanford-based project aims to safely turn on a tiny CRISPR tool inside brain cells using light-triggered control and ultrasound, potentially treating brain disorders.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project start | August 11, 2026 |
| Project end | June 30, 2031 |
| Funding year | 2026 |
| Award (this year) | $618,680 |
| NIH project number | 1R01NS142873-01A1 |
| Project ID | NIH-11451108 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with brain disorders where targeted genetic modulation could be beneficial and who are eligible for future tests or trials once safety is established.
✕Less likely to help
Early-stage preclinical work may not yet offer direct benefits to patients until safety and efficacy are confirmed in humans.
What this research studies
This project focuses on enabling precise, non-invasive gene editing in the brain by using AAV vectors that cross the blood-brain barrier and a photoswitchable CRISPR-CasMINI system. Researchers will develop a compact, light-activated CasMINI protein and lightweight, ultrasound-triggered nanotransducers to deliver the activating light deep in brain tissue. The goal is to achieve spatial and temporal control over gene editing, minimizing off-target effects while avoiding invasive procedures. Work will involve optimizing the system’s efficacy and specificity, validating light delivery methods, and demonstrating targeted gene modification in brain cells. If successful, this approach could advance basic neuroscience and pave the way for future brain disease therapies.
Why it matters
Potential benefit: If proven safe and effective, this approach could enable non-invasive, targeted gene modulation in brain cells for research and potential treatment of neurological conditions.
How similar studies have gone: Previous approaches show promise in wired or limited-scope photoregulation, but this project aims to translate that into non-invasive, deep-brain applicability with ultrasound-triggered light.
Where this research is happening
-
Stanford University
Stanford, United States Active
Researchers
- Principal investigator: Lin, Michael Z. — Stanford University
- Study coordinator: Lin, Michael Z.
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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.