Developing a new gene editing method for treating Bardet-Biedl syndrome using retinal organoids
Establishing a novel gene editing strategy for BBS7 using human retinal organoids
This study is looking at a genetic condition called Bardet-Biedl syndrome, which causes serious vision loss, and aims to fix the problem by using special lab-grown eye cells to test new treatments that could help restore vision for people with this condition.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Oregon Health & Science University NIH-funded |
| Lab location | 1 site (Portland, United States) |
| Project ID | NIH-10928808 on NIH RePORTER |
What this research studies
This research focuses on Bardet-Biedl syndrome (BBS), a genetic condition that leads to severe vision loss due to photoreceptor degeneration. The team will create a model of the disease using human retinal organoids derived from stem cells with mutations in the BBS7 gene. They will then use advanced gene editing techniques to correct these mutations and restore cell function. The effectiveness of these treatments will be evaluated in both the organoid model and in nonhuman primates to ensure safety and efficacy before potential application in humans.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Bardet-Biedl syndrome, particularly those with mutations in the BBS7 gene.
Not a fit: Patients with Bardet-Biedl syndrome caused by mutations in genes other than BBS7 may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new gene therapies that restore vision in patients with Bardet-Biedl syndrome.
How similar studies have performed: While gene editing approaches have shown promise in other conditions, this specific application for Bardet-Biedl syndrome using retinal organoids is relatively novel.
Where this research is happening
Portland, United States
- Oregon Health & Science University — Portland, United States (Active)
Researchers
- Principal investigator: Chirco, Kathleen R — Oregon Health & Science University
- Study coordinator: Chirco, Kathleen R
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.