Table 1. Non-viral vectors used to deliver genome editing agents in vivo.
Local Delivery in vivo | |||||
---|---|---|---|---|---|
Nonviral vector | Target gene | Disease | Editing Agent | Form of editing agent | Reference |
Turbofect(Polymer) | HPV Genome | Cervical cancer | TALEN | pDNA | [26] |
DNA NCs (DNA/Polymer) | GFP | - | CRISPR/Cas9 | RNP | [27] |
Polymer | Tumor suppressor genes | - | CRISPR/Cas9 | pDNA | [28] |
Lipofectamine (Lipid) | EGFP | - | CRISPR/Cas9 | RNP | [29] |
DNA-conjugated Gold/Polymer | DMD | DMD | CRISPR/Cas9 | RNP | [30] |
Lipid | TMC1 | Hearing loss | CRISPR/Cas9 | RNP | [31] |
Polymer | β-globin | - | PNA | PNA | [32] |
Polymer | CFTR | Cystic fibrosis | PNA | PNA | [33] |
Systemic Delivery in vivo | |||||
---|---|---|---|---|---|
Nonviral vector | Target gene | Disease | Editing Agent | Form of editing agent | Reference |
Lipid | HBV DNA, PCSK9 | HBV infection, hypercholesterolemia | CRISPR/Cas9 | mRNA | [34] |
Zwitterionic Amino Lipid | LoxP | - | CRISPR/Cas9 | mRNA | [35] |
Lipid | TTR | transthyretin (TTR)- mediated amyloidosis | CRISPR/Cas9 | mRNA | [36] |
Polymer | CCR5 | HIV | PNA | PNA | [37] |
Polymer | β-globin | β-thalassemia | γPNA | γPNA | [17] |