Results 71 to 80 of about 7,856 (243)

The effect of CFTR modulators on structural lung disease in cystic fibrosis

Frontiers in Pharmacology, 2023
Background: Newly developed quantitative chest computed tomography (CT) outcomes designed specifically to assess structural abnormalities related to cystic fibrosis (CF) lung disease are now available.
L. C. Mok, Antonio Garcia-Uceda, M. Cooper, M. K. D. Kemner-van de Corput, Marleen de Bruijne, N. Feyaerts, T. Rosenow, K. De Boeck, S. Stick, H. Tiddens   +9 more
semanticscholar   +1 more source

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

Molecular Therapy: Nucleic Acids
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 ...
Daichi Hinata, Yukari Kai, Ryosuke Fukuda, Yuka Kamada, Yuuya Kasahara, Kiyomi Sasaki, Tokuyuki Yoshida, Satoshi Obika, Takao Inoue, Tsukasa Okiyoneda   +9 more
doaj   +1 more source

CGM patterns in adults with cystic fibrosis-related diabetes before and after elexacaftor-tezacaftor-ivacaftor therapy

Journal of Clinical & Translational Endocrinology, 2022
Cystic fibrosis-related diabetes (CFRD) is a common complication of cystic fibrosis that is associated with worse outcomes and higher mortality rates.
Hanna Crow, Charles Bengtson, Xiaosong Shi, Leland Graves, III, Abeer Anabtawi   +4 more
doaj   +1 more source

Organoid-guided synergistic treatment of minimal function CFTR mutations with CFTR modulators, roflumilast and simvastatin: a personalised approach

European Respiratory Journal, 2023
Extract Highly effective cystic fibrosis transmembrane conductance regulator (CFTR) protein-targeting modulator therapies (HEMTs) facilitate strong clinical improvements in a large proportion of people with cystic fibrosis (CF) [1, 2]. More specifically,
S. Spelier, K. M. de Winter-de Groot, Natascha Keijzer-Nieuwenhuijze, Y. Liem, Kors van der Ent, J. Beekman, Lieke S. Kamphuis   +6 more
semanticscholar   +1 more source

Non‐pulmonary CFTR‐related symptom improvement with ivacaftor in p.Phe508del/p.Arg117His (7T) cystic fibrosis

Respirology Case Reports, 2023
Diagnosis and management of CRMS/CFSPID and cystic fibrosis (CF) with mild phenotypes remains challenging, and this extends to expanding practice with the use of CFTR modulators.
Stephanie L. Kuek, R. John H. Massie
doaj   +1 more source

Identifying transcription factors controlling the basal expression of human MRP4 highlights a substantial role for Sp1

FEBS Open Bio, EarlyView.
The MRP4 transporter exports several drugs and signaling molecules. Here, we identified key promoter elements regulating basal MRP4 expression. Using reporter assays, we defined a conserved region with essential Sp1 and contributory Ets sites, which controlled basal MRP4 expression.
Debora Singer, Angelique Kragl, Katrin Ziems, Juliane Glaubitz, Sophie Grammbauer, Susanne Hildebrandt, Mladen V. Tzvetkov, Gabriele Jedlitschky   +7 more
wiley   +1 more source

Strategies used to access CFTR modulators in countries without reimbursement agreements.

Journal of Cystic Fibrosis
CFTR modulators represent the international standard of care for the treatment of cystic fibrosis (CF). Yet due to prices of over $250,000 per year they are functionally inaccessible for people with CF (pwCF) unless reimbursed by healthcare systems ...
Jonathan Guo, G. Hennessy, B. Young, Andrew Hill   +3 more
semanticscholar   +1 more source

CFTR modulator use and risk of nontuberculous mycobacteria positivity in cystic fibrosis, 2011–2018

ERJ Open Research, 2022
Background People with cystic fibrosis are at increased risk of pulmonary nontuberculous mycobacteria (NTM) disease. Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are associated with reduced lung infection with pathogens like ...
Emily E. Ricotta, D. Rebecca Prevots, Kenneth N. Olivier   +2 more
doaj   +1 more source

TDP‐43 Aggregation: The Healthy‐Toxic Balance of the Prion‐Like Domain

Advanced Science, EarlyView.
TDP‐43 function relies on a delicate balance between reversible phase‐separated states and irreversible aggregation. Under physiological conditions, TDP‐43 forms dynamic droplets and oligomers that support normal cellular functions. In pathological contexts, this balance shifts toward aberrant aggregation, leading to toxic species.
Luca Zangrando, Emanuele Buratti, Francesca Paron   +2 more
wiley   +1 more source

The Ubiquitin Ligase RNF34 Participates in the Peripheral Quality Control of CFTR (RNF34 Role in CFTR PeriQC)

Frontiers in Molecular Biosciences, 2022
The peripheral protein quality control (periQC) system eliminates the conformationally defective cystic fibrosis transmembrane conductance regulator (CFTR), including ∆F508-CFTR, from the plasma membrane (PM) and limits the efficacy of pharmacological ...
Shogo Taniguchi, Yukiko Ito, Hibiki Kiritani, Asuka Maruo, Ryohei Sakai, Yuji Ono, Ryosuke Fukuda, Tsukasa Okiyoneda   +7 more
doaj   +1 more source