Results 51 to 60 of about 5,785 (196)

Impact of Airway Inflammation on the Efficacy of CFTR Modulators

Cells, 2021
Defective CFTR biogenesis and activity in cystic fibrosis airways leads to airway dehydration and impaired mucociliary clearance, resulting in chronic airway infection and inflammation.
Carla M. P. Ribeiro, Martina Gentzsch
doaj   +1 more source

Pregnancy‐related effect on elexacaftor, tezacaftor and ivacaftor pharmacokinetics in women with cystic fibrosis

British Journal of Clinical Pharmacology, EarlyView.
Aim The number of pregnancies among women with cystic fibrosis (wwCF) has steadily increased over the past decade. However, the pharmacokinetics (PK) of elexacaftor–tezacaftor–ivacaftor (ETI) during gestation remains uncharacterized, despite its widespread use in this population.
Paulette Magnas, Naim Bouazza, Frantz Foissac, Saïk Urien, Léo Froelicher‐Bournaud, Gabrielle Lui, Victoria Guillemart, Nicolas Carlier, Jennifer Da Silva, Johanna Fesenbeckh, Reem Kanaan, Isabelle Honoré, Clémence Martin, Jean‐Marc Treluyer, Vassilis Tsatsaris, Pierre‐Régis Burgel, Sihem Benaboud   +16 more
wiley   +1 more source

Tobramycin pharmacokinetic and pharmacodynamic targets in people with cystic fibrosis

British Journal of Clinical Pharmacology, EarlyView.
Intravenous tobramycin is a first‐line treatment for Pseudomonas aeruginosa infection in people with cystic fibrosis (CF). Tobramycin exhibits concentration‐dependent activity; however, excess drug exposure can lead to nephrotoxicity and ototoxicity. While dosing typically targets serum peak (Cmax) and trough (Cmin) concentrations, the area under the ...
Kiera H. Harwood, Sarah Bannister, Stephen Duffull, April Martinez, Sarath Ranganathan, Shivanthan Shanthikumar, Amanda Gwee   +6 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

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

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

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

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