Table 2.
Comparison of the different gene vectors for neurodegenerative disorders.
Vector type | Specific characteristics | Advantage | Disadvantage |
---|---|---|---|
AAVs | Numerous AAV serotypes; lack of targeting and site-specific; relative safety profile | Relative stable transgene expression; nonpathogenic; various serotypes available | Immune responses; limited gene packaging capacity |
Adv | Adv cannot introduce its gene into the host genome | Lower genotoxicity and insertional mutagenesis | Immune responses; relative transient transgene expression; re-administration; requires receptors for cell uptake |
LVs | Retroviruses can integrate DNA payloads into the host genome | Lower frequency of administration; stable and long-term transgene expression | Immune responses; genotoxicity; insertional mutagenesis |
Polymer- and lipid-based vectors | Non-viral vectors can be altered to impart desired functionalities | Large-scale production; controlled release; large gene packaging capacity; lower immunogenicity | Cytotoxicity; nonspecific binding and rapid clearance |