Controlled removal of a nonviral episomal vector from transfected cells

  • Rupprecht S
  • Hagedorn C
  • Seruggia D
 et al. 
  • 26


    Mendeley users who have this article in their library.
  • 12


    Citations of this article.


An ideal vector to be used in gene therapy should allow long-term and regulated expression of the therapeutic sequence, but in many cases, it would be most desirable to remove all ectopic vector sequences from the cell once expression is no longer required. The vector pEPI is the first nonviral autonomous replicon that was constructed for mammalian cells. It represents a minimal model system to study the epigenetic regulation of replication and transcription but is also regarded as a promising alternative to currently used viral vector systems in gene therapy. Its function relies on a transcription unit linked to an S/MAR sequence. We constructed an inducible pEPI vector system based on the Tet ON system in which transcription is switched on in the presence of doxycycline. We show that for vector replication and long-term maintenance an ongoing transcription running into the S/MAR element is required. Once established, the vector is lost from the cell upon switching off transcription from the gene linked to the S/MAR. This feature provides not only controlled transgene expression but also the possibility to remove all vector molecules from the cells upon demand. This inducible episomal nonviral vector system will find broad application in gene therapy but also in reprogramming of somatic cells or modification of stem cells. © 2010 Elsevier B.V.

Author-supplied keywords

  • Episomal vector
  • Gene therapy
  • Inducible expression
  • Mitotic stability

Get free article suggestions today

Mendeley saves you time finding and organizing research

Sign up here
Already have an account ?Sign in

Find this document


  • Ernst WagnerLudwig-Maximilians-Universität Munich

  • Sina Rupprecht

  • Claudia Hagedorn

  • Davide Seruggia

  • Terese Magnusson

  • Manfred Ogris

Cite this document

Choose a citation style from the tabs below

Save time finding and organizing research with Mendeley

Sign up for free