Self-amplifying RNA therapy encoding CNTF with disulfiram co-delivery promotes optic nerve repair through microglial pyroptosis inhibition and RGC axonal regeneration - PubMed
5 hours ago
- #optic nerve repair
- #RNA therapy
- #neuroinflammation
- Self-amplifying RNA therapy combined with disulfiram co-delivery promotes optic nerve repair.
- The therapy targets microglial pyroptosis inhibition and RGC axonal regeneration.
- Traumatic optic neuropathy (TON) causes irreversible vision loss with no current effective treatment.
- Microglial pyroptosis via the NLRP3/CASP1/GSDMD pathway exacerbates RGC degeneration.
- Engineered lipid nanoparticles (LNPs) deliver disulfiram and saRNA encoding CNTF.
- The combinatorial strategy suppresses neuroinflammation and provides neurotrophic support.
- Results show enhanced RGC survival, axonal regeneration, and visual function restoration.
- The approach is a promising multi-target treatment for neuroinflammatory and neurodegenerative diseases.