Fig. 1

The general process of gene transduction with a viral vector: the AAV or other viral vector inserts its single-stranded DNA into the targeted cell, and the DNA is taken up into the nucleus where it is converted into double-stranded DNA by host cell machinery. This gene, along with its accompanying promoter is inserted between inverted terminal repeats to form an episomal concatemer in the host cell nucleus. Normal transcription and translation processes take place to produce the protein product of interest. DNA is depicted by the blue coils, and RNA by red coils