Adeno-associated viral vector gene transfer into leptomeningeal xenografts.

Publication Type Academic Article
Authors Rosenfeld M, Bergman I, Schramm L, Griffin J, Kaplitt M, Meneses P
Journal J Neurooncol
Volume 34
Issue 2
Pagination 139-44
Date Published 09/01/1997
ISSN 0167-594X
Keywords Cerebellar Neoplasms, Gene Transfer Techniques, Genetic Therapy, Medulloblastoma, Meningeal Neoplasms, Subarachnoid Space
Abstract Leptomeningeal carcinomatosis is a painful and debilitating complication of cancer. Indwelling reservoirs provide continuous assess to the subarachnoid space, making leptomeningeal cancer potentially amenable to gene therapy. Adeno-associated virus (AAV) is a defective virus not associated with any human disease. We used an AAV vector to transduce medulloblastoma (DAOY) cells in a nude rat model of leptomeningeal disease. After intraventricular injection of vector carrying the bacterial lacZ gene, beta-galactosidase positive cells were found in the implanted tumor and in ependymal and subependymal cells but not in underlying normal brain parenchyma. No evidence of virally-mediated toxicity was noted in the animals. The results of this pilot study demonstrate that AAV vectors may be used to transfer and express foreign genes in established leptomeningeal tumors.
DOI 10.1023/a:1005702228721
PubMed ID 9210060
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