Design
Review
Key result
Recombinant adeno-associated virus utilizes multiple pathways for transgene expression and predominantly exists as an episome, though it has a low frequency of host genome integration.
Authors
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Low but present rAAV integration risk warrants ongoing genomic monitoring in gene therapy; leaves open precise long-term integration frequency and consequences.
This review summarizes the mechanisms of rAAV transduction, highlighting its predominantly episomal nature and low but present risk of host genome integration.
Schultz et al. (2008) conducted a review in Genetic disorders (e.g., hemophilia, cystic fibrosis, muscular dystrophies). Recombinant adeno-associated virus (rAAV) was evaluated. Recombinant adeno-associated virus utilizes multiple pathways for transgene expression and predominantly exists as an episome, though it has a low frequency of host genome integration.