Personalized CFTR Modulator Therapy for G85E and N1303K 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 CFTR mutations responsive in Fischer rat thyroid cells, including G85E, but not N1303K. However, k...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10418744/
https://www.ncbi.nlm.nih.gov/pubmed/37569738
http://dx.doi.org/10.3390/ijms241512365

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10418744/
https://www.ncbi.nlm.nih.gov/pubmed/37569738
http://dx.doi.org/10.3390/ijms241512365

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