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

Nature Genetics volume 3, pages 229–234 (1993)Cite this article

Abstract

To evaluate the potential for adenovirus–mediated central nervous system (CMS) gene transfer, the replication deficient recombinant adenovirus vectors Ad.RSVβgal (coding for β–galactosidase) and Ad–α1AT (coding for human α1–antitrypsin) were administered to the lateral ventricle of rats. Ad.RSVβgal transferred β–galactosidase to ependymal cells lining the ventricles whereas Ad–α1AT mediated α1 –antitrypsin secretion into the cerebral spinal fluid for 1 week. These observations, together with β–galactosidase activity in the globus pallidus and substantia nigra following stereotactic administration of Ad.RSVβgal to the globus pallidus, suggest that adenovirus vectors will be useful for CNS gene therapy.

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Authors and Affiliations

  1. Pulmonary Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland, 20892, USA

    Gianluigi Bajocchi, Ronald G. Crystal & Andrea Mastrangeli

  2. Section of Laboratory Animal Medicine and Surgery, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland, 20892, USA

    Sanford H. Feldman

Authors
  1. Gianluigi Bajocchi
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  2. Sanford H. Feldman
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  3. Ronald G. Crystal
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  4. Andrea Mastrangeli
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Bajocchi, G., Feldman, S., Crystal, R. et al. Direct in vivo gene transfer to ependymal cells in the central nervous system using recombinant adenovirus vectors. Nat Genet 3, 229–234 (1993). https://doi.org/10.1038/ng0393-229

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