Results 71 to 80 of about 1,208,918 (256)

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

Long term in vitro expansion of epithelial stem cells enabled by pharmacological inhibition of PAK1-ROCK-Myosin II and TGF-β signaling [PDF]

, 2018
Summary: Despite substantial self-renewal capability in vivo, epithelial stem and progenitor cells located in various tissues expand for a few passages in vitro in feeder-free condition before they succumb to growth arrest.
Challberg, Sharon S, Lee, Hyung Joo, McQuiston, Travis J, Pollok, Brian A, Shrivastava, Anura, Wang, Ruipeng, Wang, Ting, Zhang, Chengkang   +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

Effects of CFTR modulators on serum biomarkers of liver fibrosis in children with cystic fibrosis

medRxiv, 2022
The cystic fibrosis transmembrane conductance regulator (CFTR) corrector/potentiator combinations lumacaftor/ivacaftor and elexacaftor/tezacaftor/ivacaftor improve sweat chloride, pulmonary function, and nutrition. Yet it is unclear whether they may also
Steven Levitte, Y. Fuchs, Russell Wise, Zachary M. Sellers   +3 more
semanticscholar   +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

A survey: Understanding the health and perspectives of people with CF not benefiting from CFTR modulators

Pediatric Pulmonology, 2022
While the advent of cystic fibrosis transmembrane conductance regulator (CFTR) modulator use has improved daily life and long‐term prognosis of CF for many with approved CFTR mutations, approximately 10% of people with CF (pwCF) have only symptomatic ...
Emily Kramer-Golinkoff, Amanda Camacho, Liza Kramer, J. Taylor-Cousar   +3 more
semanticscholar   +1 more source

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

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

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  

CFTR Modulators Rescue the Activity of CFTR in Colonoids Expressing the Complex Allele p.[R74W;V201M;D1270N]/dele22_24

International Journal of Molecular Sciences, 2023
An Italian, 46-year-old female patient carrying the complex allele p.[R74W;V201M;D1270N] in trans with CFTR dele22_24 was diagnosed at the Cystic Fibrosis (CF) Center of Verona as being affected by CF-pancreatic sufficient (CF-PS) in 2021.
K. Kleinfelder, Elena Somenza, A. Farinazzo, Jessica Conti, V. Lotti, R. V. Latorre, L. Rodella, A. Massella, F. Tomba, Marina Bertini, C. Sorio, P. Melotti   +11 more
semanticscholar   +1 more source