Table 2.
Approaches for in vivo delivery of gene editors.
Viral Delivery | Pros | Cons |
---|---|---|
AAVs | Minimal risk of integration into the host genome Mild host immune responses Can be pseudotyped |
Limited size of the cargo that can be packaged Continuous expression of editors, increasing risk of off-target effects Limited possibility to redose, due to the immune response |
AdVs | Minimal risk of integration Not limited by the size of cargo |
Undesirable immune responses Undesirable side effects Not suited for targeted delivery Continuous expression of editors, increasing risk of off-target effects |
Lentiviruses | Can be pseudotyped Not limited by the size of cargo |
Integrated into the host genome Continuous expression of editors, increasing risk of off-target effects |
Non-viral delivery | ||
LNPs | Minimal safety and immunogenicity concerns Possibility of re-dosing Can be targeted to specific cell populations Transient expression of editors, thus minimizing off-target effects |
Low transfection efficiency |
AdVs, adenoviruses; AAVs, adeno-associated viruses; LNPs, lipid-based nanoformulations.