High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes

J Virol. 2006 Jun;80(12):6192-4. doi: 10.1128/JVI.00526-06.

Abstract

Adeno-associated virus (AAV) vectors are being considered for in vivo applications of gene therapy in the treatment of a variety of disorders. This study evaluates the biology of second-generation vectors based on the novel serotypes AAV7 and AAV8 and containing self-complementary genomes in the nonhuman primate liver. Stable levels of transgene expression were achieved in cynomolgus macaques and suggest efficiencies at least 2 log higher than what could be achieved with AAV2 vectors using traditional single-stranded genomes. Analysis of DNAs from tissues revealed high levels of vector in the liver that appeared proportional to the relative amounts of transgene expression.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • DNA, Complementary
  • Dependovirus / genetics*
  • Gene Expression
  • Genetic Therapy
  • Genetic Vectors
  • Genome, Viral / genetics*
  • Liver / metabolism*
  • Liver / virology*
  • Macaca fascicularis
  • Serotyping
  • Transgenes / genetics*

Substances

  • DNA, Complementary