Article
Expanding AAV Packaging Capacity with Trans-splicing or Overlapping Vectors: A Quantitative Comparison
1 Oct 2001
Abstract excerpt
Recombinant adeno-associated (rAAV) viral vectors hold great therapeutic potential for human diseases. However, a relatively small packaging capacity (less than 5 kb) has limited the application of rAAV for certain diseases such as cystic fibrosis and Duchenne muscular dystrophy. Here we compared two mechanistically distinct approaches to overcome packaging restraints with rAAV vectors. The trans-splicing...
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