Targeting the E1 ubiquitin-activating enzyme (UBA1) improves elexacaftor/tezacaftor/ivacaftor efficacy towards F508del and rare misfolded CFTR mutants

The advent of Trikafta (Kaftrio in Europe) (a triple-combination therapy based on two correctors—elexacaftor/tezacaftor—and the potentiator ivacaftor) has represented a revolution for the treatment of patients with cystic fibrosis (CF) carrying the most common misfolding mutation, F508del-CFTR. This...

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Detalles Bibliográficos
Autores principales: Borgo, Christian, D’Amore, Claudio, Capurro, Valeria, Tomati, Valeria, Sondo, Elvira, Cresta, Federico, Castellani, Carlo, Pedemonte, Nicoletta, Salvi, Mauro
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8924136/
https://www.ncbi.nlm.nih.gov/pubmed/35292885
http://dx.doi.org/10.1007/s00018-022-04215-3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8924136/
https://www.ncbi.nlm.nih.gov/pubmed/35292885
http://dx.doi.org/10.1007/s00018-022-04215-3

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