Aim
To design and test a hybrid nanocarrier with the capacity to lower Huntington's Disease gene (HTT) expression and prevent or diminish inflammation.
Background
Nanocarriers loaded with siRNA can be administered intranasally to provide a noninvasive, safe alternative to direct intracerebral or intrathecal infusions. Dual-function nanocarriers can also be designed to deliver several payloads that address different components of the pathological process.
Conclusion
A novel hybrid nanocarrier system with dual actions is effective in lowering HTT gene expression and attenuating inflammatory processes.
Methods
Novel hybrid nanoparticles were fabricated using a chitosan-based matrix core loaded with siRNA and an outer shell consisting of a lipid composition containing cannabidiol.
Results
Incubation of hybrid nanoparticles in mesenchymal stem cell cultures obtained from a YAC128 transgenic mouse modeling Huntington's disease resulted in effective lowering of mutant HTT gene expression and reduced levels of expression of the proinflammatory cytokine IL-6.