Using a dual CRISPR approach to treat vision and hearing loss in older adults
Dual CRISPR/Cas9 exon deleting approach for the treatment of USH2A-associated disorders
This study is testing a new gene therapy to help people over 65 who are dealing with both vision and hearing loss, aiming to reduce inflammation and improve the health of their eyes and ears.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts Eye and Ear Infirmary NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-10702163 on NIH RePORTER |
What this research studies
This research investigates a novel treatment for dual sensory loss, which includes age-related vision loss and hearing loss in individuals over 65. The approach utilizes a dual CRISPR/Cas9 technique to delete specific exons associated with USH2A-related disorders, aiming to reduce inflammation and neurodegeneration caused by complement dysregulation. By targeting the complement alternative pathway, the study seeks to improve the health of retinal and auditory tissues. Patients may receive gene therapy that delivers fusion proteins to enhance the body's ability to manage inflammation and promote tissue repair.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals aged 65 and older who are experiencing age-related macular degeneration and hearing loss.
Not a fit: Patients who are younger than 65 or do not have age-related vision or hearing loss may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to improved vision and hearing outcomes for older adults suffering from dual sensory loss.
How similar studies have performed: Previous research has shown success with similar gene therapy approaches in animal models, indicating potential for efficacy in human applications.
Where this research is happening
Boston, United States
- Massachusetts Eye and Ear Infirmary — Boston, United States (Active)
Researchers
- Principal investigator: Parsons, Nathaniel Bryan David — Massachusetts Eye and Ear Infirmary
- Study coordinator: Parsons, Nathaniel Bryan David
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.