Generation of a tumor- and tissue-specific episomal non-viral vector system.

ABSTRACT:

Background

A key issue for safe and reproducible gene therapy approaches is the autologous and tissue-specific expression of transgenes. Tissue-specific expression in vivo is either achieved by transfer vectors that deliver the gene of interest into a distinct cell type or by use of tissue-specific expression cassettes. Here we present the generation of non-viral, episomally replicating vectors that are able to replicate in a tissue specific manner thus allowing tissue specific transgene expression in combination with episomal replication. The episomal replication of the prototype vector pEPI-1 and its derivatives depends exclusively on a transcription unit starting from a constitutively active promoter extending into the scaffold/matrix attachment region (S/MAR).

Results

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SUBMITTER: Haase R 

PROVIDER: S-EPMC3728224 | biostudies-literature | 2013 Jun

REPOSITORIES: biostudies-literature