Personalized CFTR Modulator Therapy for <i>G85E</i> and <i>N1303K</i> Homozygous Patients with Cystic Fibrosis

Personalized CFTR Modulator Therapy for <i>G85E</i> and <i>N1303K</i> Homozygous Patients with Cystic Fibrosis

CFTR modulator therapy with elexacaftor/tezacaftor/ivacaftor (ETI) has been approved for people with CF and at least one F508del allele in Europe. In the US, the ETI label has been expanded to 177 rare <i>CFTR</i> mutations responsive in Fischer rat thyroid cells, including <i>G85E...

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Bibliographic Details
Main Authors: Simon Y. Graeber, Anita Balázs, Niklas Ziegahn, Tihomir Rubil, Constanze Vitzthum, Linus Piehler, Marika Drescher, Kathrin Seidel, Alexander Rohrbach, Jobst Röhmel, Stephanie Thee, Julia Duerr, Marcus A. Mall, Mirjam Stahl
Format: Article
Language:English
Published: MDPI AG 2023-08-01
Series:International Journal of Molecular Sciences
Subjects:
cystic fibrosis
CFTR
CFTR modulator
G85E
N1303K
human nasal epithelial cells
Online Access:https://www.mdpi.com/1422-0067/24/15/12365

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https://www.mdpi.com/1422-0067/24/15/12365

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