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Article

Personalized CFTR Modulator Therapy for G85E and N1303K Homozygous Patients with Cystic Fibrosis.

International journal of molecular sciences - 2 Aug 2023

Graeber Simon Y, Balázs Anita, Ziegahn Niklas, Rubil Tihomir, Vitzthum Constanze, Piehler Linus, Drescher Marika, Seidel Kathrin, Rohrbach Alexander, Röhmel Jobst, Thee Stephanie, Duerr Julia, Mall Marcus A, Stahl Mirjam

Abstract excerpt

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 CFTR mutations responsive in Fischer rat thyroid cells, including G85E, but not N1303K. However, knowledge on the effect of ETI on G85E or N1303K CFTR function remains limited. In vitro effects of ETI were measured...

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Benzodioxoles
Chlorides
Cystic Fibrosis
Cystic Fibrosis Transmembrane Conductance Regulator

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 37569738
DOI: 10.3390/ijms241512365
PMCID: PMC10418744

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Personalized CFTR Modulator Therapy for G85E and N1303K Homozygous Patients with Cystic Fibrosis.PMID 37569738Read ↗

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