Antisense oligonucleotide targeting the E3 ligase RFFL potentiates CFTR modulator efficacy in CF primary bronchial epithelial cells

Cystic fibrosis (CF) is most commonly caused by the ΔF508 mutation in the CFTR gene, leading to misfolding and degradation of the CFTR protein. Although CFTR modulators such as elexacaftor/tezacaftor/ivacaftor (ETI) provide clinical benefit, their efficacy is limited, particularly in patients with r...

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Autors principals:	Daichi Hinata, Yukari Kai, Ryosuke Fukuda, Yuka Kamada, Yuuya Kasahara, Kiyomi Sasaki, Tokuyuki Yoshida, Satoshi Obika, Takao Inoue, Tsukasa Okiyoneda
Format:	Artigo
Idioma:	Inglês
Publicat:	Elsevier 2025-12-01
Col·lecció:	Molecular Therapy: Nucleic Acids
Matèries:	MT: Oligonucleotides: Therapies and Applications CFTR cystic fibrosis transmembrane conductance regulator RFFL ASO antisense oligonucleotide
Accés en línia:	http://www.sciencedirect.com/science/article/pii/S2162253125003105
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