Adeno associated virus in human gene therapy
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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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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



Raav construction

1 KB

EcoRI

BglII

BglII

XhoI

BglII

BglII

MDR1 cDNA

3’ ITR

5’ ITR

CMV promoter

SV40 PolyA

rAAV Construction




Conclusions
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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