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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Presentation Transcript
slide2
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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