Efficient repetitive gene delivery to skeletal muscle using recombinant adenovirus vector containing the Coxsackievirus and adenovirus receptor cDNA

Gene Ther. 2001 Jan;8(1):20-7. doi: 10.1038/sj.gt.3301359.

Abstract

To improve adenovirus-mediated gene delivery to skeletal muscle, we have used a recombinant adenovirus vector encoding the human Coxsackievirus and adenovirus receptor (hCAR). Because CAR is expressed at a lower level in rodent myoblasts and muscle fibers than in other tissues, we expected that elevated expression of CAR in skeletal muscle would improve the efficacy of adenovirus-mediated gene transfer. Since the mouse myoblasts, C2C12 cells, showed low sensitivity to infection by recombinant adenovirus 5, we initially infected these cells at a high multiplicity of infection (MOI) of 250 with the recombinant adenovirus containing hCAR cDNA and LacZ gene. Subsequent infection by recombinant adenovirus containing the marker gene, green fluorescence protein, became efficient even at a low MOI of 25. Thus, elevated hCAR expression in mouse muscle fibers made a second virus inoculation at low doses possible. We also demonstrated that the elevated hCAR expression did not influence muscle membrane integrity. Our results suggest that co-expression of CAR and a therapeutic gene by adenovirus vector constitutes a novel strategy to advance gene therapy for hereditary muscle diseases.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenoviridae / genetics*
  • Animals
  • Cell Culture Techniques
  • Coxsackie and Adenovirus Receptor-Like Membrane Protein
  • Cytoskeletal Proteins / metabolism
  • DNA, Complementary / genetics
  • Dystroglycans
  • Dystrophin / metabolism
  • Enterovirus / genetics
  • Gene Transfer Techniques*
  • Genetic Vectors*
  • Humans
  • Injections, Intramuscular
  • Membrane Glycoproteins / metabolism
  • Mice
  • Mice, Inbred BALB C
  • Mice, Nude
  • Muscle, Skeletal / metabolism*
  • Receptors, Virus / genetics*
  • Receptors, Virus / metabolism
  • Transduction, Genetic

Substances

  • CLMP protein, human
  • CLMP protein, mouse
  • Coxsackie and Adenovirus Receptor-Like Membrane Protein
  • Cytoskeletal Proteins
  • DAG1 protein, human
  • DNA, Complementary
  • Dystrophin
  • Membrane Glycoproteins
  • Receptors, Virus
  • Dystroglycans