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...

ver descrição completa

Detalhes bibliográficos

Principais autores:	Daichi Hinata, Yukari Kai, Ryosuke Fukuda, Yuka Kamada, Yuuya Kasahara, Kiyomi Sasaki, Tokuyuki Yoshida, Satoshi Obika, Takao Inoue, Tsukasa Okiyoneda
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Elsevier 2025-12-01
Colecção:	Molecular Therapy: Nucleic Acids
Assuntos:	MT: Oligonucleotides: Therapies and Applications CFTR cystic fibrosis transmembrane conductance regulator RFFL ASO antisense oligonucleotide
Acesso em linha:	http://www.sciencedirect.com/science/article/pii/S2162253125003105
Tags:	Adicionar Tag Sem tags, seja o primeiro a adicionar uma tag!