Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors

Nat Biotechnol. 2000 May;18(5):527-32. doi: 10.1038/75390.

Abstract

A major shortcoming to the use of adeno-associated virus (rAAV) vectors is their limited packaging size. To overcome this hurdle, we split an expression cassette and cloned it into two separate vectors. The vectors contained either a nuclear localizing Escherichia coli lacZ transgene (nlslacZ) with a splice acceptor, or the human elongation factor 1alpha ( EF1alpha) gene enhancer/promoter(s) (EF1alphaEP) with a splice donor. We co-injected a promoter-less nlslacZ vector with a vector containing either a single EF1alphaEP or a double copy of the EF1alphaEP in a head-to-head orientation, into the portal vein of mice. Gene expression, measured by both transduction efficiency and quantitation of the recombinant protein, was as much as 60-70% of that obtained from mice that received a single vector containing a complete EFalphaEP/nlslacZ expression cassette. This two-vector approach may allow development of gene therapy strategies that will carry exogenous DNA sequences with large therapeutic cDNAs and/or regulatory elements.

Publication types

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

MeSH terms

  • Animals
  • Dependovirus / genetics*
  • Genetic Therapy / methods
  • Genetic Vectors / administration & dosage*
  • Injections, Intravenous
  • Lac Operon
  • Liver / virology
  • Mice
  • Peptide Elongation Factor 1 / biosynthesis
  • Peptide Elongation Factor 1 / genetics
  • Portal Vein
  • Recombinant Proteins / biosynthesis*
  • Recombination, Genetic*
  • Transgenes
  • beta-Galactosidase / biosynthesis
  • beta-Galactosidase / genetics

Substances

  • Peptide Elongation Factor 1
  • Recombinant Proteins
  • beta-Galactosidase