Cystic Fibrosis

Name: Cystic Fibrosis
Brand: LAP LAMBERT Academic Publishing
Availability: OutOfStock
Rating: 5 (1 reviews)

By Karima Gourari-Bouzouina

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Description

Cystic fibrosis (CF) is a lethal genetic disorder mainly affecting Caucasians, caused by mutations in the CFTR gene encoding a chloride ion channel. The most common ¿F508 mutation alters CFTR folding and prevents its expression on epithelial cell surfaces, disrupting ion and water transport and leading to thick mucus, a hallmark of CF lung disease. Treatments have advanced, with antibiotics managing chronic infections and CFTR modulators (ivacaftor, tezacaftor, elexacaftor) improving protein function. Yet, many patients with rare mutations or drug resistance remain untreated. Emerging therapies now focus on gene therapy to correct or replace the defective CFTR gene and on cell therapy to regenerate functional epithelium from corrected stem cells. Combining these strategies offers a promising path toward personalized, long-term correction of the molecular cause of CF.

Product details

Binding:: Paperback
Number of Pages:: 100
Release Date:: 2025-10-31
Publication Date:: 2025-10-31
Publisher:: LAP LAMBERT Academic Publishing
Languages:: Original: English
ISBN10:: 6209166717
ISBN13:: 9786209166716
Weight:: 167 g
Height:: 150 cm
Width:: 220 cm
Thickness:: 6 cm

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