Gene therapy for a type of inherited vision loss
Official title: Translational Gene Therapy for CNGB1 Retinitis Pigmentosa
Funded at Michigan State University · NIH-10368093 · Data updated 2026-11-12
This study is working on a new gene therapy to help people with a specific type of vision loss called autosomal recessive retinitis pigmentosa, which is caused by changes in the CNGB1 gene, by using a special virus to deliver the correct gene to the eye and hopefully improve vision and protect the eye from further damage.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Michigan State University NIH-funded |
| Lab location | 1 site (East Lansing, United States) |
| Project start | April 1, 2018 |
| Project end | July 31, 2025 |
| Funding year | 2022 |
| Award (this year) | $1,517,749 |
| NIH project number | 5R24EY027285-05 |
| Project ID | NIH-10368093 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with autosomal recessive retinitis pigmentosa linked to CNGB1 mutations.
✕Less likely to help
Patients with other forms of retinitis pigmentosa or those without CNGB1 mutations may not benefit from this research.
What this research studies
This research focuses on developing a gene therapy approach for treating autosomal recessive retinitis pigmentosa (arRP) caused by mutations in the CNGB1 gene. The project aims to optimize a viral vector that can deliver the correct gene to the affected rod photoreceptors in the retina. By using animal models that mimic the human condition, the researchers will assess the effectiveness of this therapy in restoring vision and preventing further degeneration of retinal cells. The ultimate goal is to prepare for a clinical trial that could bring this innovative treatment to patients.
Why it matters
Potential benefit: If successful, this research could provide a new treatment option for patients suffering from a currently untreatable form of inherited vision loss.
How similar studies have gone: Previous research has shown promising results with gene therapy approaches in similar retinal conditions, indicating potential for success in this novel application.
Where this research is happening
-
Michigan State University
East Lansing, United States Active
Researchers
- Principal investigator: Petersen-Jones, Simon — Michigan State University
- Study coordinator: Petersen-Jones, Simon
Related research
Other NIH-funded projects on the same topics.
-
Inflammation in MERTK-related retinitis pigmentosa
This project aims to understand whether losing the MERTK gene causes retinal inflammation that leads to vision loss in people with MERTK-related retinitis pigmentosa.
-
Enhancing cone survival in RP through targeted cell-specific CRISPR therapy
A patient-friendly look at a new approach to protect cone cells in retinitis pigmentosa using precise gene editing to support vision.
-
Oral, once-daily MC16 medicine for retinitis pigmentosa
A gene-mutation-agnostic pill (MC16) is being studied to help people with retinitis pigmentosa by addressing a common cellular problem in the eye.
-
Role of IPM Proteoglycans in Retinal Health and Inherited Blindness
This grant studies how specific retinal extracellular matrix proteins (IMPG1 and IMPG2) affect eye health and contribute to retinitis pigmentosa, with potential implications for people who have or are at risk for inherited blindness.
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NAC Attack: a late-stage trial for preserving vision in RP patients
A large, multi-site study is testing whether taking a daily NAC pill can help people with retinitis pigmentosa keep more of their vision.
-
Non-invasive biomarker for mitochondria health in retinitis pigmentosa
Aims to test a new, non-invasive OCT-based biomarker to explain and predict how mitochondria function affects cone cells in people with retinitis pigmentosa.
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.