Somatische Gentherapie von blutbildenden Stammzellen

Zusammenfassung

Ziel der somatischen Gentherapie ist die Korrektur von Mutationen, die zu einer Erkrankung führen. Die Behandlung blutbildender Stammzellen ist dabei von besonderem klinischen Interesse, da deren Transplantation im Idealfall mit einer einzigen Behandlung zu einer dauerhaften Heilung führt. In diesem Übersichtsartikel stellen wir den Status quo zur Gentherapie von hämatopoetischen Stamm- und Vorläuferzellen dar und gehen auf neue Entwicklungen, wie z. B. den induzierten pluripotenten Stammzellen (iPS-Zellen), näher ein. 

Schlüsselwörter

hämatopoetische Stammzellen - pluripotente Stammzellen - somatische Gentherapie - Retroviren - Vektoren - homologe Rekombination

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