Strategien für die Lebergentherapie

  • V. Sandig
  • C. Hofmann
  • P. Löser
  • G. Jennings
  • G. Cichon
  • P. Schlag
  • M. Strauss
Conference paper

Zusammenfassung

Die genetische Therapie von genetischen, malignen und infektiösen Erkrankungen der Leber ist in der experimentellen Erprobung. Die gentechnischen Voraussetzungen für derartige neue Therapieformen werden gegenwärtig entwickelt. Als entscheidend für den künftigen Erfolg wird sich die Qualität der Genübertragungssysteme erweisen. Gegenwärtig stehen Vektoren, die von Retroviren oder Adenoviren abgeleitet sind im Mittelpunkt des Interesses. Alternativ wird v.a. der Weg über spezifische Rezeptoren der Leber erschlossen. Alle existierenden Systeme sind bisher nur für eine Anwendung ex vivo geeignet. Ein erster Versuch zur Therapie einer genetischen Erkrankung der Leber durch Gentransfer in Hepatozyten der Patienten und nachfolgende Transplantation der Hepatozyten hat die Grenzen eines derartigen Verfahrens aufgezeigt. Die Lösung kann nur in der Entwicklung einer In-vivo-Strategie bestehen. Der Beitrag gibt eine kurze Übersicht der existierenden Transfersysteme und diskutiert eigene neue Ansätze, die auf eine In-vivo-Anwendung abzielen.

Summary

Genetic therapy of genetic, malignant and infectious diseases of the liver is currently being established in experimental systems. Gene technological prerequisits for this new type of therapy are being developed. The quality of transfer systems will be of crucial importance for future success. Currently, vectors derived from retroviruses and adenoviruses are in the center of interest. However, alternative ways via liver-specific receptors are also explored. All existing delivery systems are only useful for ex vivo application so far. An initial trial for gene transfer into hepatocytes and subsequent transplantation of the treated hepatocytes back into the patient has clearly shown the limits of this kind of approach. Therefore, the only solution for the problem is the development of in vivo transfer strategies. This article provides a brief summary of the existing delivery systems and discusses new approaches of our own aiming at in vivo application.

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Copyright information

© Springer-Verlag Berlin Heidelberg 1995

Authors and Affiliations

  • V. Sandig
  • C. Hofmann
  • P. Löser
  • G. Jennings
  • G. Cichon
  • P. Schlag
  • M. Strauss

There are no affiliations available

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