Article
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.
International journal of molecular sciences - 19 Jun 2023
Kleinfelder Karina, Villella Valeria Rachela, Hristodor Anca Manuela, Laudanna Carlo, Castaldo Giuseppe, Amato Felice, Melotti Paola, Sorio Claudio
Abstract excerpt
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 compounds in recovering their molecular defects. Here we used both rectal organoids (colonoids) and primary nasal...
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