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Overview of gene therapy vectors. |
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| Genetic material |
Packaging capacity |
Vector genome forms |
Advantages |
Disadvantages |
|
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|
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| Retrovirus |
RNA |
8 kb |
Integrated |
- High efficiency integration |
- Transduction only in dividing cells |
| - No viral immune response |
- Insertional carcinogenesis |
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| - Long-term expression |
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| Lentivirus |
RNA |
8 kb |
Integrated |
- Non-dividing cells |
- Integration into active genes |
| - Long-term expression |
- Risk of replication competent HIV |
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| Adenovirus |
dsDNA |
Up to 35 kb (HDAd) |
Episomal |
- Non-dividing cells |
- Acute toxicity |
| - Large cloning capacity |
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| - High transduction levels |
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| - Long-term expression (HDAd) |
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| Adeno-associated vectors |
ssDNA |
5–9 kb |
Episomal (> 90%) |
- Non-dividing cells |
- Limited cloning capacity |
| Integrated (< 10%) |
- Long-term expression |
- CTL-mediated immune reaction |
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| Naked plasmid DNA |
dsDNA |
Unlimited |
Episomal |
- Non dividing cells |
- Low efficiency of transduction |
| - No inflammatory response |
- Efficient and clinically relevant delivery method still to be developed |
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| - Large cloning capacity |
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| - Long-term expression |
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| - Ease preparation |
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dsDNA = double stranded DNA; ssDNA = single stranded DNA; HDAd = helper-dependent adenoviral vector; CTL = cytotoxic T lymphocyte. | |||||
Brunetti-Pierri Italian Journal of Pediatrics 2008 34:2 doi:10.1186/1824-7288-34-2 |
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