Table 1 Gene therapy vectors
Class | Main advantages | Key limitations |
|---|---|---|
Non-viral | not infectious, not toxic | low efficacy, short-term transgene expression |
Adenoviral | high efficacy | immunogenic, short-term transgene expression |
Retro−/lentiviral | long-term transgene expression | risk of insertional mutagenesis, restricted host-range, only for dividing cells (retroviral vectors), HIV-based material (lentiviral vectors) |
rAAV | high efficacy, long-term transgene expression, also for quiescent cells | complex to prepare, size limitation |