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J Genet Syndr Gene Ther. 2013 Apr 29;4(133). doi: 10.4172/2157-7412.1000133.

Improved Adeno-associated Viral Gene Transfer to Murine Glioma.

Journal of genetic syndromes & gene therapy

I Zolotukhin, D Luo, Os Gorbatyuk, Be Hoffman, Kh Warrington, Rw Herzog, Jk Harrison, O Cao

Affiliations

  1. Department of Pediatrics, University of Florida, Gainesville, FL, USA.

PMID: 24319629 PMCID: PMC3852404 DOI: 10.4172/2157-7412.1000133

Abstract

Glioblastoma (GBM) is a deadly primary brain tumor. Current treatment, consisting of surgical removal of the tumor mass followed by chemotherapy and/or radiotherapy, does not significantly prolong survival. Gene therapies for GBM are being developed in clinical trials, for example using adenoviral vectors. While adeno-associated virus (AAV) represents an alternative vector system, limited gene transfer to glioma cells has hampered its use. Here, we evaluated newly emerged variants of AAV capsid for gene delivery to murine glioma. We tested a mutant AAV2 capsid devoid of 3 surface-exposed tyrosine residues, AAV2 (Y444-500-730F), and a "shuffed" capsid (ShH19, containing sequences from several serotypes) that had previously been selected for enhanced glial gene delivery. AAV2 (Y-F) and ShH19 showed improved transduction of murine glioma GL261 cells

Keywords: AAV; CNS; Cancer; Gene transfer; Glioma

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