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Zystische Fibrose by Karima Gourari-Bouzouina

Zystische Fibrose

by Karima Gourari-Bouzouina , Zahia Boucherit-Otmani
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Book cover type: Paperback
  • ISBN13: 9786209303920
  • Binding: Paperback
  • Subject: N/A
  • Publisher: Verlag Unser Wissen
  • Publisher Imprint: Verlag Unser Wissen
  • Publication Date:
  • Pages: 112
  • Original Price: GBP 52.98
  • Language: German
  • Edition: N/A
  • Item Weight: 159 grams
  • BISAC Subject(s): Life Sciences / Biology

Zystische Fibrose (CF) ist eine t�dliche Erbkrankheit, die vorwiegend Menschen kaukasischer Abstammung betrifft und durch Mutationen im CFTR-Gen, das f�r einen Chloridionenkanal kodiert, verursacht wird. Die h�ufigste Mutation, ∆F508, ver�ndert die Faltung des CFTR-Proteins und verhindert dessen Expression auf der Oberfl�che von Epithelzellen. Dies st�rt den Ionen- und Wassertransport und f�hrt zu z�hem Schleim, einem charakteristischen Merkmal der CF-Lungenerkrankung. Die Behandlungsm�glichkeiten haben sich verbessert: Antibiotika werden zur Behandlung chronischer Infektionen eingesetzt, und CFTR-Modulatoren (Ivacaftor, Tezacaftor, Elexacaftor) verbessern die Proteinfunktion. Dennoch bleiben viele Patienten mit seltenen Mutationen oder Arzneimittelresistenz unbehandelt. Neue Therapieans�tze konzentrieren sich nun auf die Gentherapie, um das defekte CFTR-Gen zu korrigieren oder zu ersetzen, sowie auf die Zelltherapie, um funktionsf�higes Epithel aus korrigierten Stammzellen zu regenerieren. Die Kombination dieser Strategien bietet einen vielversprechenden Weg zu einer personalisierten, langfristigen Korrektur der molekularen Ursache von CF.

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