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Kinder- und Jugendmedizin 2022; 22(01): 47-55
DOI: 10.1055/a-1677-7644
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Gentherapie bei angeborenen Defekten der Immunität

Sujal Ghosh
1   Klinik für Kinder-Onkologie, -Hämatologie und Klinische Immunologie, Zentrum für Kinder- und Jugendmedizin, Universitätsklinikum Düsseldorf
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ZUSAMMENFASSUNG

Seit 50 Jahren werden angeborene Defekte der Immunität erfolgreich durch eine allogene hämatopoetische Stammzelltransplantation behandelt. Neben verschiedenen Faktoren, wie z. B. Grunderkrankungen und Komorbiditäten, ist die geeignete Spenderverfügbarkeit von Bedeutung. Die Spender-gegen-Empfänger-Erkrankung ist gerade im HLA-nicht-angepassten Setting eine wesentliche Ursache für Morbidität und Mortalität. Seit über 30 Jahren ist man bestrebt, für diese seltenen Erkrankungen gentherapeutische Konzepte zu entwickeln. Der schwere kombinierte Immundefekt, die chronisch-septische Granulomatose und das Wiskott-Aldrich-Syndom zählen zu den ersten genetischen Erkrankungen, die mittels viraler Vektoren durch ein Genadditionsverfahren behandelt werden konnten. Lehren aus diesen Studien konnten auch für eine Reihe nicht-immunologischer Erkrankungen gezogen werden. Überschattet wurden die initial erfolgversprechenden Behandlungsergebnisse mit dem häufigen Auftreten von Leukämien. Modifikationen viraler Vektoren durch zahlreiche Sicherheitsmechanismen sind nun Bestandteil moderner Gentherapiestudien, sodass mittlerweile diese Plattform für eine Reihe weiterer Erkrankung zur Verfügung steht.



Publication History

Article published online:
25 February 2022

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