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Table 1 Overview of gene therapy vectors.

From: Gene therapy for inborn errors of liver metabolism: progress towards clinical applications

  Genetic material Packaging capacity Vector genome forms Advantages Disadvantages
Retrovirus RNA 8 kb Integrated - High efficiency integration - Transduction only in dividing cells
     - No viral immune response - Insertional carcinogenesis
     - Long-term expression  
Lentivirus RNA 8 kb Integrated - Non-dividing cells - Integration into active genes
     - Long-term expression - Risk of replication competent HIV
Adenovirus dsDNA Up to 35 kb (HDAd) Episomal - Non-dividing cells - Acute toxicity
     - Large cloning capacity  
     - High transduction levels  
     - Long-term expression (HDAd)  
Adeno-associated vectors ssDNA 5–9 kb Episomal (> 90%) - Non-dividing cells - Limited cloning capacity
    Integrated (< 10%) - Long-term expression - CTL-mediated immune reaction
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
     - Large cloning capacity  
     - Long-term expression  
     - Ease preparation  
  1. dsDNA = double stranded DNA; ssDNA = single stranded DNA; HDAd = helper-dependent adenoviral vector; CTL = cytotoxic T lymphocyte.