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In vivo gene gun-mediated transduction into rat heart with Epstein-Barr virus-based episomal vectors
1Department of Surgery III, Nara Medical University, Kashihara, Japan. nszk@naramed-u.ac.jp
Background:
Gene guns have been used to transfer genes into various organs, but there has been no report of successful gene gun-mediated gene transfer into the heart. In this study, we assessed the possibility of gene therapy using a gene gun and an episomal plasmid vector.
Methods:
Gene transfer was performed using two sizes of gold particles and two plasmids (an episomal vector and a conventional plasmid vector). From the first to eighth week after the bombardment, rats were sacrificed. The excised hearts were subjected to X-gal staining and histologic examination. To ensure that plasmid was not distributed to organs other than the heart, the presence of the beta-gal sequence was examined by polymerase chain reaction analyses.
Results:
Gene expression persisted for 6 weeks. The episomal vector apparently contributed to long-lasting expression. Infiltration of monocytes or leukocytes was very faint. The beta-gal DNA was detected in bombarded hearts but not other organs.
Conclusions:
Gene gun-mediated transfer of the episomal vector into beating heart may provide a simple, efficient, and useful strategy for gene therapy.