AAV vectors design/construction/QC/extraction

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AAV vectors design strategies:

Single strain AAV (ssAAV)

      The typical AAV genome is the encapsidation of a single-stranded DNA (ssDNA) genome. In the case of AAV, the separate plus or minus polarity strands are packaged with equal frequency, and either is infectious. In gene therapy application with rAAV, the virus transduces the cell with a single stranded DNA (ssDNA) flanked by two Inverted Terminal Repeats (ITRs). These ITRs form hairpins configuration at the end of the sequence to serve as primers for cellular DNA polymerase to synthesize the complementary strand in host cells before subsequent steps of infection.

Self-complementary AAV (scAAV)

     The coding region of scAAV has been designed to form an intra-molecular double-stranded DNA template, in host cells, two complementary halves of scAAV will associate to form one double stranded DNA (dsDNA) unit that is ready for immediate replication and transcription, which avoid cell mediated synthesis of the second strand. Usually scAAV delivery faster and more robust gene expression compared with ssAAV, as a disadvantage, scAAV can only hold half of the coding amount (~2.4kb) instead of the full coding capacity in ssAAV (~4.7 kb).

Both of ssAAV and ScAAV are widely used for gene therapy, please select the right rAAV vector according to your project aims.