Results 61 to 70 of about 12,522 (293)

The therapeutic potential of small-molecule modulators of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel [PDF]

, 2014
The cystic fibrosis transmembrane conductance regulator (CFTR) plays a pivotal role in fluid and electrolyte movements across ducts and tubes lined by epithelia.
Cai, Zhiwei, Cami-Kobeci, Gerta, Husbands, Stephen, Khuituan, Pissared, Li, Hongyu, Liu, Jia, Sheppard, David, Wang, Yiting   +7 more
core   +2 more sources

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

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

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

CFTR Modulators Restore Acidification of Autophago-Lysosomes and Bacterial Clearance in Cystic Fibrosis Macrophages

Frontiers in Cellular and Infection Microbiology, 2022
Cystic fibrosis (CF) human and mouse macrophages are defective in their ability to clear bacteria such as Burkholderia cenocepacia. The autophagy process in CF (F508del) macrophages is halted, and the underlying mechanism remains unclear.
Asmaa Badr, Asmaa Badr, Mostafa Eltobgy, Kathrin Krause, Kathrin Krause, Kaitlin Hamilton, Shady Estfanous, Shady Estfanous, Kylene P. Daily, Arwa Abu Khweek, Arwa Abu Khweek, Ahmad Hegazi, Midhun N. K. Anne, Cierra Carafice, Frank Robledo-Avila, Youssra Saqr, Xiaoli Zhang, Tracey L. Bonfield, Mikhail A. Gavrilin, Santiago Partida-Sanchez, Stephanie Seveau, Estelle Cormet-Boyaka, Amal O. Amer   +22 more
doaj   +1 more source

Human pluripotent stem cell-derived acinar/ductal organoids generate human pancreas upon orthotopic transplantation and allow disease modelling [PDF]

, 2016
Objective The generation of acinar and ductal cells from human pluripotent stem cells (PSCs) is a poorly studied process, although various diseases arise from this compartment.
Antony, Justin S., Breunig, Markus, Costa, Ivan G., Eiseler, Tim, Hermann, Patrick C., Hohwieler, Meike, Illing, Anett, Kleger, Alexander, Kleiderman, Susanne M., Kormann, Michael, Kuo, Chao Chung, Lechel, André, Leichsenring, Michael, Liebau, Stefan, Lin, Qiong, Mayer, Tobias, Mayerle, Julia, Müller, Martin, Perkhofer, Lukas, Renz, Susanne, Rosendahl, Jonas, Sainz, Bruno, Sendler, Matthias, Seufferlein, Thomas, Stockmann, Marianne, Wagner, Martin, Zenke, Martin, Zenker, Martin   +27 more
core   +2 more sources

Nanomolar-potency 'co-potentiator' therapy for cystic fibrosis caused by a defined subset of minimal function CFTR mutants. [PDF]

, 2019
Available CFTR modulators provide no therapeutic benefit for cystic fibrosis (CF) caused by many loss-of-function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel, including N1303K. We previously introduced the
Finkbeiner, Walter E, Haggie, Peter M, Nielson, Dennis W, Phuan, Puay-Wah, Rivera, Amber A, Tan, Joseph-Anthony, Verkman, Alan S, Zlock, Lorna   +7 more
core  

Proceedings: Regenerative Medicine for Lung Diseases: A CIRM Workshop Report. [PDF]

, 2017
The mission of the California Institute of Regenerative Medicine (CIRM) is to accelerate treatments to patients with unmet medical needs. In September 2016, CIRM sponsored a workshop held at the University of California, Los Angeles, to discuss ...
DeWitt, Natalie D, Gomperts, Brigitte, Kadyk, Lisa C   +2 more
core   +1 more source

Strategies to Improve the Lipophilicity of Hydrophilic Macromolecular Drugs

Advanced Healthcare Materials, EarlyView.
Hydrophilic macromolecular drugs can be successfully lipidized by covalent attachment of lipids, by hydrophobic ion pairing with negatively or positively charged surfactants, and by dry or wet reverse micelle formation. Lipophilicity enhancement of hydrophilic macromolecules has several benefits including stability and bioavailability improvement ...
Sera Lindner, Stefan Keim, Soheil Haddadzadegan, Odile Fernandez Romero, Katrin Zöller, Gabriel Stern, Ilaria Cesi, Krum Kafedjiiski, Andreas Bernkop‐Schnürch   +8 more
wiley   +1 more source

Rapid therapeutic advances in CFTR modulator science [PDF]

Pediatric Pulmonology, 2018
AbstractCystic fibrosis (CF) is an autosomal recessive genetic disease caused by variants in the gene encoding the cystic fibrosis transmembrane conduction regulator (CFTR) protein. Loss of CFTR function disrupts chloride, bicarbonate and regulation of sodium transport, producing a cascade of mucus obstruction, inflammation, pulmonary infection, and ...
openaire   +2 more sources