Results 91 to 100 of about 7,856 (243)

Outcomes of pregnancy in women with cystic fibrosis (CF) taking CFTR modulators - an international survey.

, 2020
BACKGROUND As their long-term prognosis improves, women with CF are increasingly choosing to have children, but the safety of CFTR modulators in pregnancy and breastfeeding is currently unknown.
Taylor-Cousar, Jennifer L, Nash, Edward F, Middleton, Peter G   +2 more
core   +1 more source

Modulators of CFTR protein function in the treatment of cystic fibrosis - a literature review

Journal of Education, Health and Sport, 2022
Cystic fibrosis (CF) is a multi-system genetic disease with an autosomal recessive inheritance mechanism. The breakthroughs in the therapy of patients with CF turned out to be modulators of CFTR protein function.
Dariusz Chojęta, Monika Mierzwa, Hubert Wróblewski, Aleksandra Zimna, Ewelina Zygmunt, Anna Kozłowska, Katarzyna Łysiak   +6 more
doaj   +1 more source

A descriptive case series of hepatotoxicity associated with CFTR modulators and possible relevance of pharmacogenetic polymorphisms in cystic fibrosis patients

British Journal of Clinical Pharmacology, EarlyView.
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are widely used in patients with cystic fibrosis and significantly improve respiratory function and quality of life. However, their effectiveness may be limited by liver damage, which sometimes leads to treatment discontinuation, and the mechanisms underlying this remain poorly ...
Clara Laffitte Redondo, Venceslas Bourdin, Abd El Kader Ait Tayeb, Sihem Benaboud, Inès Martins, Caroline Joyau, Myriam Benhamida, Isabelle Sermet‐Gaudelus, Clémence Martin, Pierre Régis Burgel, Céline Verstuyft, Laurent Chouchana, Estelle Ayme‐Dietrich   +12 more
wiley   +1 more source

RFFL-mediated protein quality control limits functional rescue of TRID-CFTR modulator combination therapy for cystic fibrosis nonsense mutations

Cellular and Molecular Life Sciences
Cystic fibrosis (CF) is a monogenic disorder caused by mutations in the CFTR gene, which encodes a cAMP-regulated anion channel at the apical plasma membrane (PM) of epithelial cells. CFTR modulators have recently been approved as effective therapies for
Hazuki Tateishi, Yukako Doi, Yuka Kamada, Tsukasa Okiyoneda   +3 more
doaj   +1 more source

CFTR Modulator Response in Nasal Organoids Derived from People with Cystic Fibrosis

Cells
Despite the progressive extension of CFTR variant eligibility to the triple combination of elexacaftor/tezacaftor/ivacaftor (ETI), most rare CFTR pathogenic variants remain ineligible for CFTR modulators.
Stefania Lo Cicero, Germana Castelli, Aurora Ceci, Anna Maria Cerio, Giovanna Blaconà, Mariarita Virgulti, Sara Allushi, Giovanni Sette, Francesca Spadaro, Felice Amato, Paola Melotti, Claudio Sorio, Giuseppe Cimino, Mauro Biffoni, Marco Lucarelli, Adriana Eramo   +15 more
doaj   +1 more source

Targeting neutrophil extracellular traps in metabolic and immune niche: Nanomaterials for diabetes tissue regeneration

BMEMat, EarlyView.
The effects of NETs on regeneration of various diabetic tissues, and strategies targeting NETs for diabetes tissue regeneration. In the diabetic environment, NETs undergo complex metabolic and immune reprogramming, leading to dynamic changes in antibacterial and proinflammatory functions, and affecting regeneration of multiple systemic tissues.
Xinyi Jiang, Muxin Yue, Kai Mao, Boon Chin Heng, Noor Azlin Yahya, Yunsong Liu, Zheng Li   +6 more
wiley   +1 more source

Status and future of recombinant adeno‐associated virus vector manufacturing

Biotechnology Progress, EarlyView.
Abstract Sixty years of adeno‐associated virus (AAV) research illustrates a trajectory marked by basic science exploration, iterative innovation, persistent challenges, a number of clinical setbacks, as well as commercial therapeutic triumphs. This continual evolution has led to recombinant AAV (rAAV) becoming a cornerstone of modern gene therapy ...
Frank Agbogbo, David Dismuke
wiley   +1 more source

Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two F508del alleles

ERJ Open Research, 2019
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator gene (CFTR) that result in diminished quantity and/or function of the CFTR anion channel.
Jennifer L. Taylor-Cousar, Marcus A. Mall, Bonnie W. Ramsey, Edward F. McKone, Elizabeth Tullis, Gautham Marigowda, Charlotte M. McKee, David Waltz, Samuel M. Moskowitz, Jessica Savage, Fengjuan Xuan, Steven M. Rowe   +11 more
doaj   +1 more source

Setting the stage for CFTR modulator studies in infants [PDF]

Thorax, 2014
Because irreversible damage to the cystic fibrosis (CF) lung is already observed in infancy, a need has arisen to assess and monitor lung function early in life. Infant lung function testing (ILFT) is non-invasive, does not require radiation or anaesthesia and can be repeated frequently; therefore, development, standardisation and validation of ...
openaire   +3 more sources

Impact of cystic fibrosis transmembrane conductance regulator modulator therapies on liver stiffness and liver enzymes: An observational perspective single‐center cohort study

JPGN Reports, EarlyView.
Abstract Objectives The efficacy of cystic fibrosis transmembrane conductance regulator (CFTR)‐modulator therapies in preventing or ameliorating cystic fibrosis liver disease (CFLD) by correcting CFTR in cholangiocytes is not well‐documented. This study aimed to assess liver function during CFTR‐modulators.
Laura Giugliano, Elisabetta Augustoni, Antonio Pizzol, Anna Opramolla, Cristina Chiadò, Anna Zagaria, Irene Esposito, Giulia Roberto, Alice Ponte, Andrea Evangelista, Paola Quarello, Michele Pinon, Pier Luigi Calvo   +12 more
wiley   +1 more source