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Molekulare Grundlagen des Gentransfers und Anwendung für die Gentherapie

  • Karsten Brand
  • Michael Strauss
Part of the Handbuch der Molekularen Medizin book series (HDBMOLEK, volume 2)

Zusammenfassung

Gentherapie ist das gezielte Einbringen eines funktionsfähigen Gens in Körperzellen mit therapeutischer Zielstellung. Der Gentherapeut versucht entweder eine defekte Zellfunktion wiederherzustellen, wie etwa bei der Substitutionstherapie monogenetischer Erkrankungen, oder aber der Zelle eine zusätzliche Funktion zu vermitteln, wie etwa bei der Übertragung toxischer Gene. Damit können prinzipiell alle Erkrankungen, bei denen Proteine eine entscheidende pathogenetische Rolle spielen oder eine therapeutische Funktion haben, Ziel einer gentherapeutischen Behandlung sein. Inwieweit die Gentherapie tatsächlich sinnvoll zum Einsatz kommt, richtet sich danach, ob der Umweg über das Gen gegenüber konventionellen Verfahren eher Voroder Nachteile hat. Ausgehend von teilweise beeindruckenden tierexperimentellen Ergebnissen wurden in den letzten 5 Jahren bisher 136 klinische Therapiestudien, darunter 90 zur Behandlung von Krebs, durchgeführt (Stand: März 1996). Bei bisher etwa 600 behandelten Patienten traten, von einer publizierten Ausnahme abgesehen [Crystal et al. 1994], keine gravierenden Nebenwirkungen auf. Allerdings sind bisher auch keine konsistenten Therapieerfolge beschrieben worden. Damit wird wohl erst in den nächsten Jahren zu rechnen sein, wenn die noch unausge- reifte gentherapeutische Methodik bis zur klinischen Reife weiterentwickelt worden ist.

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© Springer-Verlag Berlin Heidelberg 1998

Authors and Affiliations

  • Karsten Brand
  • Michael Strauss

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