Theratyping of the Rare CFTR Genotype A559T in Rectal Organoids and Nasal Cells Reveals a Relevant Response to Elexacaftor (VX-445) and Tezacaftor (VX-661) Combination

Theratyping of the Rare CFTR Genotype A559T in Rectal Organoids and Nasal Cells Reveals a Relevant Response to Elexacaftor (VX-445) and Tezacaftor (VX-661) Combination

Despite the promising results of new CFTR targeting drugs designed for the recovery of F508del- and class III variants activity, none of them have been approved for individuals with selected rare mutations, because uncharacterized CFTR variants lack information associated with the ability of these c...

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Bibliographic Details
Main Authors: Karina Kleinfelder, Valeria Rachela Villella, Anca Manuela Hristodor, Carlo Laudanna, Giuseppe Castaldo, Felice Amato, Paola Melotti, Claudio Sorio
Format: Article
Language:English
Published: MDPI AG 2023-06-01
Series:International Journal of Molecular Sciences
Subjects:
rectal organoids
CFTR modulators
nasal cells
rare mutations
cystic fibrosis
theratyping
Online Access:https://www.mdpi.com/1422-0067/24/12/10358

Internet

https://www.mdpi.com/1422-0067/24/12/10358

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