New approaches towards ex vivo and in vivo gene therapy

Hansjörg Hauser, D. Spitzer, E. Verhoeyen, J. Unsinger, D. Wirth

Research output: Contribution to journalReview articlepeer-review

9 Scopus citations


A number of hurdles have to be overcome for efficient and specific gene therapy approaches. Here, we report on two different strategies that should lead to an improvement of current protocols. A strategy is presented to tag unique chromosomal integration sites by means of retroviral infection, which can be reused for exchange with the gene of interest by action of site- specific recombinases. Targeting exchange is achieved in one step with 100% efficiency by a stringent positive selection, which makes further screening superfluous. With this strategy a predictable gene expression is obtained for foreign genes integrated into a predefined chromatin structure. A second approach aims at the stabilization of mouse retroviruses towards human serum which is a prerequisite for in vivo gene therapy protocols. To stabilize murine leukemia virus-based retroviruses against human serum, complement regulatory proteins were fused to the retroviral ENV proteins. This resulted in infectious and human complement-protected particles. Copyright (C) 2000 S. Karger AG, Basel.

Original languageEnglish
Pages (from-to)75-80
Number of pages6
JournalCells Tissues Organs
Issue number2-3
StatePublished - Jan 1 2000


  • Complement resistance
  • Gene targeting
  • Gene therapy
  • Retrovirus
  • Stem cells


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