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Adeno-Associated Virus in Human Gene Therapy

Adeno-Associated Virus in Human Gene Therapy. Nick Juty 6 th Sept. 2007. AAV2. Replication deficient Serotypes with tissue specificity Site specific integration chr 19 No pathogenic / immunogenic effect Can infect quiescent and dividing cells Limited capacity ( 4.6kb )

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Adeno-Associated Virus in Human Gene Therapy

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  1. Adeno-Associated Virus in Human Gene Therapy Nick Juty 6th Sept. 2007

  2. AAV2 • Replication deficient • Serotypes with tissue specificity • Site specific integration chr 19 • No pathogenic / immunogenic effect • Can infect quiescent and dividing cells • Limited capacity (4.6kb) • Clinical trials (I and II) for Cystic Fibrosis, haemophilia, Parkinson’s, arthritis

  3. Pluripotent stem cell

  4. 1 KB EcoRI BglII BglII XhoI BglII BglII MDR1 cDNA 3’ ITR 5’ ITR CMV promoter SV40 PolyA rAAV Construction

  5. P-Glycoprotein expression

  6. Dye-exclusion assay

  7. Conclusions • AAV transfection and transduction • Site-specific Integration into cell’s DNA • Expression from integrated gene • Presence of P-glycoprotein on cell surface • Extrusion of cytotoxic drugs and dye

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