Summary
This project aims to develop and test an AAV-based Retinoic Acid signaling activator to preserve cone photoreceptors in mouse models of Retinitis Pigmentosa, and to elucidate the underlying molecular mechanisms.
Elucidating the molecular mechanisms of secondary cone degeneration in models of Retinitis Pigmentosa
US · IL
NIH
grant
awarded
#nih-1R01EY038190-01
What they want
The project involves developing a novel AAV-based Retinoic Acid signaling activator, testing its ability to improve photopic visual acuity in multiple mouse models of Retinitis Pigmentosa, and investigating the molecular and cellular mechanisms downstream of this signaling pathway that contribute to cone survival. The ultimate goal is to find a gene-agnostic therapeutic option for RP patients.
Deliverables
- A novel AAV-based Retinoic Acid signaling activator
- Data on improved photopic visual acuity in mouse models of RP
- Elucidation of molecular and cellular mechanisms of cone survival via Retinoic Acid signaling
Technical requirements
- AAV-based Retinoic Acid signaling activator development
- Use of mouse models of Retinitis Pigmentosa
- Assessment of photopic visual acuity
- Molecular and cellular mechanism elucidation techniques
