MicroRNA-124 loaded lipid nanoparticles for the treatment of spinal cord injury
(2026)
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- Abstract
- Spinal cord injury (SCI) is a devastating neurological condition that results in severe impairments of motor and sensory function. MicroRNA-124 (miR-124) is a potent regulator of neuroinflammation that promotes microglial polarization toward an anti-inflammatory M2-like phenotype and supports neural regeneration. However, its clinical translation has been hindered by the lack of safe and efficient delivery systems capable of targeting the injured spinal tissue. This PhD thesis focuses on developing a new delivery system for miR-124 for SCI treatment. We used lipid nanoparticles (LNPs) to deliver miR-124 combining the selective organ targeting (SORT) strategy to improve the therapeutic effects in SCI. In the first part, we compared six ionizable lipids and selected the lipid offering the best compromise between therapeutic efficiency and lack of microglial activation. We then demonstrated the therapeutic potential of our formulation in the context of lipopolysaccharides-induced neuroinflammation. In the second part, we developed and assessed SORT-LNPs for the enhanced delivery of miR-124 in the injured lesions and therapeutic efficacy following SCI mice. Overall, our findings provide useful insights into the development of novel targeting LNPs approach acting to delivery miRNA for SCI, as a versatile platform for exploring gene therapies targeting neuroinflammation.
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Citations
Ma, Z. (2026). MicroRNA-124 loaded lipid nanoparticles for the treatment of spinal cord injury. https://hdl.handle.net/2078.5/273461