Direct in vivo gene transfer to ependymal cells in the central nervous system using recombinant adenovirus vectors Academic Article uri icon


MeSH Major

  • Adenoviruses, Human
  • Brain
  • Cerebral Ventricles
  • Ependyma
  • Genes, Bacterial
  • Transfection
  • alpha 1-Antitrypsin
  • beta-Galactosidase


  • To evaluate the potential for adenovirus-mediated central nervous system (CNS) gene transfer, the replication deficient recombinant adenovirus vectors Ad.RSV beta gal (coding for beta-galactosidase) and Ad-alpha 1AT (coding for human alpha 1-antitrypsin) were administered to the lateral ventricle of rats. Ad.RSV beta gal transferred beta-galactosidase to ependymal cells lining the ventricles whereas Ad-alpha 1AT mediated alpha 1-antitrypsin secretion into the cerebral spinal fluid for 1 week. These observations, together with beta-galactosidase activity in the globus pallidus and substantia nigra following stereotactic administration of Ad.RSV beta gal to the globus pallidus, suggest that adenovirus vectors will be useful for CNS gene therapy.

publication date

  • March 26, 1993



  • Academic Article



  • eng

Digital Object Identifier (DOI)

  • 10.1038/ng0393-229

PubMed ID

  • 8485578

Additional Document Info

start page

  • 229

end page

  • 34


  • 3


  • 3