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Verfasst von:Gentzsch, Martina [VerfasserIn]   i
 Mall, Marcus A. [VerfasserIn]   i
Titel:Ion channel modulators in cystic fibrosis
Verf.angabe:Martina Gentzsch, and Marcus A. Mall
E-Jahr:2018
Jahr:8 May 2018
Umfang:11 S.
Fussnoten:Gesehen am 02.08.2019
Titel Quelle:Enthalten in: Chest
Ort Quelle:Amsterdam : Elsevier, 1935
Jahr Quelle:2018
Band/Heft Quelle:154(2018), 2, Seite 383-393
ISSN Quelle:1931-3543
Abstract:Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and remains one of the most common life-shortening genetic diseases affecting the lung and other organs. CFTR functions as a cyclic adenosine monophosphate-dependent anion channel that transports chloride and bicarbonate across epithelial surfaces, and disruption of these ion transport processes plays a central role in the pathogenesis of CF. These findings provided the rationale for pharmacologic modulation of ion transport, either by targeting mutant CFTR or alternative ion channels that can compensate for CFTR dysfunction, as a promising therapeutic approach. High-throughput screening has supported the development of CFTR modulator compounds. CFTR correctors are designed to improve defective protein processing, trafficking, and cell surface expression, whereas potentiators increase the activity of mutant CFTR at the cell surface. The approval of the first potentiator ivacaftor for the treatment of patients with specific CFTR mutations and, more recently, the corrector lumacaftor in combination with ivacaftor for patients homozygous for the common F508del mutation, were major breakthroughs on the path to causal therapies for all patients with CF. The present review focuses on recent developments and remaining challenges of CFTR-directed therapies, as well as modulators of other ion channels such as alternative chloride channels and the epithelial sodium channel as additional targets in CF lung disease. We further discuss how patient-derived precision medicine models may aid the translation of emerging next-generation ion channel modulators from the laboratory to the clinic and tailor their use for optimal therapeutic benefits in individual patients with CF.
DOI:doi:10.1016/j.chest.2018.04.036
URL:Bitte beachten Sie: Dies ist ein Bibliographieeintrag. Ein Volltextzugriff für Mitglieder der Universität besteht hier nur, falls für die entsprechende Zeitschrift/den entsprechenden Sammelband ein Abonnement besteht oder es sich um einen OpenAccess-Titel handelt.

Volltext ; Verlag: https://doi.org/10.1016/j.chest.2018.04.036
 Volltext: http://www.sciencedirect.com/science/article/pii/S0012369218306640
 DOI: https://doi.org/10.1016/j.chest.2018.04.036
Datenträger:Online-Ressource
Sprache:eng
Sach-SW:cystic fibrosis
 pharmacotherapy
 translating basic research
K10plus-PPN:1670485978
Verknüpfungen:→ Zeitschrift

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