Abstract

Two major problems limiting the use of non-viral gene therapy include the low efficiency of gene transfer seen with non-viral vectors and limited methods for cell-specific targeting and expression. We and others have shown that translocation of plasmid DNA into the nucleus is an important rate-limiting step in gene transfer that affects transgene expression levels in non-dividing cells. Further, we have identified several DNA sequences that mediate the nuclear import of plasmids in non-dividing cells, thereby acting as DNA nuclear targeting sequences.

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