Helping children with Lowe syndrome see better
CRISPR Based Rescue of Glaucoma in Lowe Syndrome
This research explores a new gene-editing method to help children with Lowe syndrome who are at risk of losing their vision due to glaucoma.
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 ID | NIH-11081645 on NIH RePORTER |
What this research studies
Children with Lowe syndrome often develop cataracts and glaucoma, which can lead to blindness, and currently, there are no specific treatments for this condition. This project is developing a new way to use gene-editing technology to correct the underlying problem in Lowe syndrome. By targeting specific genes, the goal is to restore balance in the cells and prevent vision loss. Researchers are using human stem cells and animal models to test these new approaches.
Who could benefit from this research
Good fit: This research is focused on understanding and treating Lowe syndrome, particularly in children aged 0-11 years old who are affected by the condition.
Not a fit: Patients without Lowe syndrome or those whose vision loss is due to other causes would not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this work could lead to the first effective treatment to prevent blindness in children with Lowe syndrome.
How similar studies have performed: This project is developing a novel gene-editing approach for Lowe syndrome, as there are currently no therapies for this condition.
Where this research is happening
Stanford, United States
- Stanford University — Stanford, United States (Active)
Researchers
- Principal investigator: Sun, Yang — Stanford University
- Study coordinator: Sun, Yang
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.