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Die Entdeckung des Zystische-Fibrose-Transmembran-Regulator-(CFTR-)Gens im Jahr 1989 gab Grund zur Hoffnung, dass eine kausale Therapie der Mukoviszidose (CF) in Kürze verfügbar sein würde. Obwohl Wissenschaftler Fortschritte in Bezug auf die Korrektur des zugrundeliegenden Defekts auf Genebene erreicht haben, gibt es bislang keine verfügbare Therapie mit diesem Ansatz. Neue mutationsspezifische Therapiekonzepte...