Results 121 to 130 of about 1,459,532 (261)

The PROSPECT Is Bright for CFTR Modulators

Annals of the American Thoracic Society, 2021
Kristina Montemayor, Noah Lechtzin
openaire   +2 more sources

Nasal cells as a bronchial cell surrogate for pre-clinical assessment of drug response in cystic fibrosis

Frontiers in Pharmacology
Patient-derived airway cell cultures are used in personalized medicine strategies for people with cystic fibrosis (pwCF) to predict potential clinical response to cystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs. While bronchial
Malina Barillaro, Julie Avolio, Sharaniyaa Balachandran, Claire Bartlett, Wan Ip, Hong Ouyang, Wenming Duan, Joseph Zabner, Shaf Keshavjee, Christine Bear, Christine Bear, Theo J. Moraes, Theo J. Moraes, Tanja Gonska, Tanja Gonska   +14 more
doaj   +1 more source

Ubiquitin and ubiquitin‐like modifications in the endoplasmic reticulum stress response

The FEBS Journal, EarlyView.
Endoplasmic reticulum (ER) stress activates various proteostasis control processes, including the unfolded protein response, ribosome‐associated quality control, and ER‐associated degradation. Ubiquitin and ubiquitin‐like modifications dynamically regulate these processes to determine cell fate, promoting adaptation or inducing cell death.
Tony Avril, Matthieu Le Gallo, Elodie Lafont   +2 more
wiley   +1 more source

CFTR Function Restoration upon Elexacaftor/Tezacaftor/Ivacaftor Treatment in Patient-Derived Intestinal Organoids with Rare CFTR Genotypes. [PDF]

, 2023
Cystic fibrosis (CF) is caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator ( CFTR) gene. The combination of the CFTR modulators elexacaftor, tezacaftor, and ivacaftor (ETI) enables the effective rescue of CFTR function in ...
Muilwijk, Danya, Lefferts, Juliet W, Sonneveld van Kooten, Heleen N, Beekman, Jeffrey M, Bierlaagh, Marlou C, Janssens, Hettie M, Kroes, Suzanne, van der Ent, Cornelis K, Niemöller, Paul J, Verburg, Tibo F, van Beuningen, Sam F B, Nieuwenhuijze, Natascha D A   +11 more
core  

Effect of Cystic Fibrosis Transmembrane Conductance Regulator Modulators on Liver Enzymes Among Patients With Cystic Fibrosis: A Systematic Review and Meta-Analysis

Gastro Hep Advances
Backgrounds and Aims: This paper seeks to use existing literature to investigate the use of Cystic Fibrosis Transmembrane Conductance (CFTR) modulators and their safety and effects on the liver enzymes of cystic fibrosis (CF) patients with and without ...
Tahne Vongsavath, Kyaw Min Tun, Dwaipayan Mukhopadhyay, Chun-Han Lo, Ashok Singh, Rajan Amin, Patrick Twohig, Sammy Saab, Vignan Manne   +8 more
doaj   +1 more source

Effect of CFTR Modulators on Oxidative Stress and Autophagy in Non-CFTR-Expressing Cells

International Journal of Molecular Sciences
The triple combination therapy for cystic fibrosis (CF), including elexacaftor, tezacaftor and ivacaftor (ETI or Trikafta), has been shown to improve lung function and reduce pulmonary exacerbations, thereby enhancing the quality of life for most CF patients.
Scialò, Filippo, Cernera, Gustavo, Polise, Lorenza, Castaldo, Giuseppe, Amato, Felice, Villella, Valeria Rachela   +5 more
openaire   +3 more sources

Triple combination cystic fibrosis transmembrane receptor modulator effects on glycaemia and insulin kinetics in cystic fibrosis with and without diabetes

Internal Medicine Journal, EarlyView.
Abstract Background Greater insight into the effects of cystic fibrosis (CF) transmembrane modulators such as elexacaftor‐tezacaftor‐ivacaftor (ETI) on glucose metabolism can support a more dynamic and individualised approach to CF‐related dysglycaemia.
Yi W. Chen, Rachel Prasad, Peter G. Middleton, D. Jane Holmes‐Walker   +3 more
wiley   +1 more source

What Is a Paediatrician? Reflection on the Specialty of Paediatrics

Journal of Paediatrics and Child Health, EarlyView.
ABSTRACT A paediatrician is a specialist medical practitioner committed to the health and well‐being of infants, children and young people. However, curricula for training in paediatrics are predominantly process and content focussed, with some emphasis on professional behaviours, but because of their length do not distil the essence of what it is to ...
John Massie
wiley   +1 more source

Modulator-refractory cystic fibrosis: Defining the scope and challenges of an emerging at-risk population

Therapeutic Advances in Respiratory Disease
Cystic fibrosis (CF) causes life-shortening respiratory and systemic disease due to dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel.
Lindsay Somerville, Larry Borish, Imre Noth, Dana Albon   +3 more
doaj   +1 more source

CFTR function is impaired in a subset of patients with pancreatitis carrying rare CFTR variants [PDF]

Background: Many affected by pancreatitis harbor rare variants of the cystic fibrosis (CF) gene, CFTR, which encodes an epithelial chloride/bicarbonate channel. We investigated CFTR function and the effect of CFTR modulator drugs in pancreatitis patients
Sorio, Claudio, Frulloni, Luca, Kleinfelder, Karina, De Marchi, Giulia, Angyal, Dora, Surace, Cecilia, Alghisi, Federico, de Jonge, Hugo R., Bijvelds, Marcel J.C.; id_orcid, Groeneweg, Tessa A., Tomba, Francesco, Bernardoni, Laura, Bijvelds, Marcel J. C., De Angelis, Paola, de Pretis, Nicolò, Melotti, Paola, Rodella, Luca, Pintani, Emily, Lucidi, Vincenzina, Ciciriello, Fabiana   +19 more
core   +1 more source