Gene editing to correct BBS7-related vision loss using lab-grown human retina tissue
Establishing a novel gene editing strategy for BBS7 using human retinal organoids
['FUNDING_OTHER'] · OREGON HEALTH & SCIENCE UNIVERSITY · NIH-11115824
Researchers will use gene-editing tools to repair BBS7 mutations that lead to vision loss in people with Bardet-Biedl syndrome.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | OREGON HEALTH & SCIENCE UNIVERSITY (nih funded) |
| Locations | 1 site (PORTLAND, UNITED STATES) |
| Trial ID | NIH-11115824 on ClinicalTrials.gov |
What this research studies
This project grows human retinal organoids — lab-made mini-retinas from patient-derived stem cells — that carry BBS7 mutations. Scientists will apply prime editing delivered with lipid nanoparticles to try to permanently fix the faulty BBS7 gene in those organoids and watch whether photoreceptor cells recover. The most promising gene-editing approaches will then be tested in nonhuman primates to check dosing, immune reactions, and how well the editing tool reaches photoreceptors. The overall aim is to develop a precise, durable therapy that could one day be given to people whose vision is threatened by BBS7 mutations.
Who could benefit from this research
Good fit: Ideal candidates are people with Bardet-Biedl syndrome whose vision loss is caused specifically by disease-causing variants in the BBS7 gene.
Not a fit: People whose retinal degeneration is due to other genes, late-stage disease with lost photoreceptors, or non-genetic causes of vision loss are unlikely to benefit from this work.
Why it matters
Potential benefit: If successful, this could lead to a long-lasting therapy that restores photoreceptor function and slows or reverses vision loss from BBS7-related disease.
How similar studies have performed: AAV-based gene replacement has helped some inherited retinal diseases, but using prime editing in human retinal organoids and lipid nanoparticles for delivery is a newer approach still being tested.
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.
Conditions: Bardet Biedel syndrome, Bardet-Biedl Syndrome