| Adeno-associated virus (AAV) |
4.5 kb |
Low immunogenicity, stable transgene expression |
High infection efficiency, broad cell tropism |
Low packaging capacity, difficulty in production |
Treatment of lipoprotein lipase deficiency patients, edit the VEGFR2 gene in retinal endothelial cells within a mouse model, single-cell CRISPR screening in mouse brain |
72–74
|
| Lentivirus (LV) |
8 kb |
Large packaging capacity, low cell cycle tendency |
High infection efficiency, large packing size, long-term gene expression, persistent gene transfer |
Long lasting expression of Cas9, potential for insertional mutagenesis |
Epigenome editing targets cytokines (TNF-α, IL-1β) in the intervertebral disc to treat degenerative disc disease and chronic low-back pain, gene editing tool delivery with a gag-only strategy, vaccine development |
75,76
|
| Adenovirus (AV) |
>8 kb |
Large packaging capacity, no integration to the host genome |
Risk of off-target effects, strong immunogenicity, limited cargo capacity for gene constructs, limited reusability, high delivery efficiency |
High immunogenicity, difficult scale production |
Induces targeted mutations in a wide range of human cells, selective targeting of vascular endothelial cells in vivo, cancer treatment, gene therapy |
77,78
|
