Table 3.
Type | Category | Strategy |
---|---|---|
Viral delivery | Toxicity | Reduction of genotoxicity using specific target sequence to avoid activation of proto-oncogene or using regulatory machinery for transgene expression |
Efficiency | Enhancement of viral tropism specific to peripheral nervous system | |
Non-viral delivery | Stability | Development of enhanced vehicle to increase the stability of oligonucleotides |
Chemical modification of oligonucleotide to increase the stability | ||
Efficiency | Development of novel chemical composition or peptide to enhance cell uptake and to facilitate endolysosomal escape or nuclear translocation | |
Specificity | Isolation of novel receptors or membrane compositions in Schwann cell or axon | |
Development of novel ligands specific to peripheral nervous system |