Table 1.
Summary form of typical in vivo delivery systems and candidates for genome editing nucleases and their expression cassette.
| Typical Delivery Systems | Assessment | Genome Editing Nuclease | Clinical Trials | |||||
|---|---|---|---|---|---|---|---|---|
| Advantages | Disadvantages | Phase | Status | Clinical Trials. Gov Identifier | Reference | |||
| AAVs | High efficiency | Low packaging capacity, cost high | ZFNs, CRISPR/Cas9 | – | – | – | [59,73,74,104] | |
| AdVs | Low off-target mutagenesis | Immunoreactivity, high cost | ZFNs | I | Completed | NCT01044654 | [49,105,106] | |
| II | Completed | NCT01252641 | ||||||
| I | Completed | NCT00842634 | ||||||
| HCAdVs | High packaging capacity | Cell-specific targeting is difficult to achieve | TALENs | – | – | – | [32] | |
| CPP, e.g., TAT-TALEN proteins; CPP-Cas9 proteins | Low off-target mutagenesis | Immunoreactivity | TALENs, CRISPR/Cas9 | – | – | – | [63,64,86] | |
| Candidates for delivering plasmids of nucleases | DOTAP-cholesterol | Easy to produce, large packaging capacity | Large particle size, low targeting efficiency, toxic | – | I/II | Active | NCT01455389 | [8] |
| PEI | Easy to produce, large packaging capacity | Low targeting efficiency, toxic | – | II | Active | NCT00595088 | ||
| PEG-PEI-Cholesterol | Easy to produce, large packaging capacity with small particle size, low toxic | Low targeting efficiency | – | II | Active | NCT01118052 | ||