Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

Gene Ther. 2001 Aug;8(16):1248-54. doi: 10.1038/sj.gt.3301514.

Abstract

Adeno-associated virus (AAV) vectors package single-stranded genomes and require host-cell synthesis of the complementary strand for transduction. However, when the genome is half wild-type size, AAV can package either two copies, or dimeric inverted repeat DNA molecules. Dimeric, or self-complementary molecules (scAAV) should spontaneously reanneal, alleviating the requirement for host-cell DNA synthesis. We generated and characterized scAAV vectors in order to bypass the rate-limiting step of second-strand synthesis. In vitro, scAAV vectors were five- to 140-fold more efficient transducing agents than conventional rAAV, with a 5.9:1 particle to transducing unit ratio. This efficiency is neither greatly increased by co-infection with Ad, nor inhibited by hydroxyurea, demonstrating that transduction is independent of DNA synthesis. In vivo, scAAV expressing erythropoietin resulted in rapid and higher levels of hematocrit than a conventional single-stranded vector. These novel scAAV vectors represent a biochemical intermediate in rAAV transduction and should provide new insights into the biology of vector transduction.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Animals
  • Cell Line
  • DNA, Complementary
  • DNA, Viral
  • Dependovirus / genetics*
  • Erythropoietin / genetics
  • Gene Expression
  • Genetic Engineering
  • Genetic Vectors / pharmacology*
  • Green Fluorescent Proteins
  • HeLa Cells
  • Hematocrit
  • Humans
  • Liver / metabolism
  • Luminescent Proteins / genetics
  • Mice
  • Mice, Inbred BALB C
  • Transduction, Genetic*

Substances

  • DNA, Complementary
  • DNA, Viral
  • Luminescent Proteins
  • Erythropoietin
  • Green Fluorescent Proteins