Cargando…

CFTR Rescue by Lumacaftor (VX-809) Induces an Extensive Reorganization of Mitochondria in the Cystic Fibrosis Bronchial Epithelium

Background: Cystic Fibrosis (CF) is a genetic disorder affecting around 1 in every 3000 newborns. In the most common mutation, F508del, the defective anion channel, CFTR, is prevented from reaching the plasma membrane (PM) by the quality check control of the cell. Little is known about how CFTR phar...

Descripción completa

Detalles Bibliográficos
Autores principales:	Braccia, Clarissa, Christopher, Josie A., Crook, Oliver M., Breckels, Lisa M., Queiroz, Rayner M. L., Liessi, Nara, Tomati, Valeria, Capurro, Valeria, Bandiera, Tiziano, Baldassari, Simona, Pedemonte, Nicoletta, Lilley, Kathryn S., Armirotti, Andrea
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9222197/ https://www.ncbi.nlm.nih.gov/pubmed/35741067 http://dx.doi.org/10.3390/cells11121938

Ejemplares similares

Journey on VX-809-Based Hybrid Derivatives towards Drug-like F508del-CFTR Correctors: From Molecular Modeling to Chemical Synthesis and Biological Assays
por: Parodi, Alice, et al.
Publicado: (2022)

Proteomics and Metabolomics for Cystic Fibrosis Research
por: Liessi, Nara, et al.
Publicado: (2020)

Distinctive lipid signatures of bronchial epithelial cells associated with cystic fibrosis drugs, including Trikafta
por: Liessi, Nara, et al.
Publicado: (2020)

Vx-809, a CFTR Corrector, Acts through a General Mechanism of Protein Folding and on the Inflammatory Process
por: Pecoraro, Michela, et al.
Publicado: (2023)

Clinical Consequences and Functional Impact of the Rare S737F CFTR Variant and Its Responsiveness to CFTR Modulators
por: Terlizzi, Vito, et al.
Publicado: (2023)

Pseudomonas aeruginosa Reduces VX-809 Stimulated F508del-CFTR Chloride Secretion by Airway Epithelial Cells
por: Stanton, Bruce A., et al.
Publicado: (2015)

Bioactive Thymosin Alpha-1 Does Not Influence F508del-CFTR Maturation and Activity
por: Armirotti, Andrea, et al.
Publicado: (2019)

Increased efficacy of VX-809 in different cellular systems results from an early stabilization effect of F508del-CFTR
por: Farinha, Carlos M, et al.
Publicado: (2015)

Targeting the E1 ubiquitin-activating enzyme (UBA1) improves elexacaftor/tezacaftor/ivacaftor efficacy towards F508del and rare misfolded CFTR mutants
por: Borgo, Christian, et al.
Publicado: (2022)

Partial Rescue of F508del-CFTR Stability and Trafficking Defects by Double Corrector Treatment
por: Capurro, Valeria, et al.
Publicado: (2021)

The L467F-F508del Complex Allele Hampers Pharmacological Rescue of Mutant CFTR by Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Patients: The Value of the Ex Vivo Nasal Epithelial Model to Address Non-Responders to CFTR-Modulating Drugs
por: Sondo, Elvira, et al.
Publicado: (2022)

High-Content Surface and Total Expression siRNA Kinase Library Screen with VX-809 Treatment Reveals Kinase Targets that Enhance F508del-CFTR Rescue
por: Perkins, Lydia A., et al.
Publicado: (2018)

New Insights into the Binding Features of F508del CFTR Potentiators: A Molecular Docking, Pharmacophore Mapping and QSAR Analysis Approach
por: Righetti, Giada, et al.
Publicado: (2020)

Discovery of a picomolar potency pharmacological corrector of the mutant CFTR chloride channel
por: Pedemonte, Nicoletta, et al.
Publicado: (2020)

Molecular Docking and QSAR Studies as Computational Tools Exploring the Rescue Ability of F508del CFTR Correctors
por: Righetti, Giada, et al.
Publicado: (2020)

Virtual Drug Repositioning as a Tool to Identify Natural Small Molecules That Synergize with Lumacaftor in F508del-CFTR Binding and Rescuing
por: Fossa, Paola, et al.
Publicado: (2022)

Activity of lumacaftor is not conserved in zebrafish Cftr bearing the major cystic fibrosis‐causing mutation
por: Laselva, Onofrio, et al.
Publicado: (2019)

Comprehensive Analysis of Combinatorial Pharmacological Treatments to Correct Nonsense Mutations in the CFTR Gene
por: Venturini, Arianna, et al.
Publicado: (2021)

Gain- and Loss-of-Function CFTR Alleles Are Associated with COVID-19 Clinical Outcomes
por: Baldassarri, Margherita, et al.
Publicado: (2022)

Spectral Analysis on Cuba-Lumacaftor: Cubane as Benzene Bioisosteres of Lumacaftor
por: Wang, Dongdong, et al.
Publicado: (2023)

Theratyping of the Rare CFTR Genotype A559T in Rectal Organoids and Nasal Cells Reveals a Relevant Response to Elexacaftor (VX-445) and Tezacaftor (VX-661) Combination
por: Kleinfelder, Karina, et al.
Publicado: (2023)

VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1
por: Ren, Hong Yu, et al.
Publicado: (2013)

Matrine in association with FD-2 stimulates F508del-cystic fibrosis transmembrane conductance regulator activity in the presence of corrector VX809
por: Marengo, Barbara, et al.
Publicado: (2017)

Treatment of Cystic Fibrosis Patients Homozygous for F508del with Lumacaftor-Ivacaftor (Orkambi(®)) Restores Defective CFTR Channel Function in Circulating Mononuclear Cells
por: Favia, Maria, et al.
Publicado: (2020)

Dual Activity of Aminoarylthiazoles on the Trafficking and Gating Defects of the Cystic Fibrosis Transmembrane Conductance Regulator Chloride Channel Caused by Cystic Fibrosis Mutations
por: Pedemonte, Nicoletta, et al.
Publicado: (2011)

Identifying the molecular target sites for CFTR potentiators GLPG1837 and VX-770
por: Yeh, Han-I, et al.
Publicado: (2019)

Lumacaftor/ivacaftor for cystic fibrosis
Publicado: (2019)

Isobaric Labeling Proteomics Allows a High-Throughput Investigation of Protein Corona Orientation
por: Liessi, Nara, et al.
Publicado: (2020)

The Autophagy Inhibitor Spautin-1 Antagonizes Rescue of Mutant CFTR Through an Autophagy-Independent and USP13-Mediated Mechanism
por: Pesce, Emanuela, et al.
Publicado: (2018)

Modeling long-term health outcomes of patients with cystic fibrosis homozygous for F508del-CFTR treated with lumacaftor/ivacaftor
por: Rubin, Jaime L., et al.
Publicado: (2019)

CFTR Modulator Therapy with Lumacaftor/Ivacaftor Alters Plasma Concentrations of Lipid-Soluble Vitamins A and E in Patients with Cystic Fibrosis
por: Sommerburg, Olaf, et al.
Publicado: (2021)

Bridging for lung transplantation with lumacaftor/ivacaftor
por: Pedersen, Søren Sperling, et al.
Publicado: (2018)

VX-770-mediated potentiation of numerous human CFTR disease mutants is influenced by phosphorylation level
por: Cui, Guiying, et al.
Publicado: (2019)

Innovative Strategy toward Mutant CFTR Rescue in Cystic Fibrosis: Design and Synthesis of Thiadiazole Inhibitors of the E3 Ligase RNF5
por: Brusa, Irene, et al.
Publicado: (2023)

WS07.06 CFTR impacts SARS-CoV-2 infection in cystic fibrosis
por: Bragonzi, A., et al.
Publicado: (2023)

Pharmacological Correctors of Mutant CFTR Mistrafficking
por: Pedemonte, Nicoletta, et al.
Publicado: (2012)

Treatment of Polarized Cystic Fibrosis Airway Cells With HGF Prevents VX-661-Rescued F508del-CFTR Destabilization Caused by Prolonged Co-exposure to VX-770
por: Matos, Ana M., et al.
Publicado: (2021)

Inferring differential subcellular localisation in comparative spatial proteomics using BANDLE
por: Crook, Oliver M., et al.
Publicado: (2022)

Corticosteroid use and increased CXCR2 levels on leukocytes are associated with lumacaftor/ivacaftor discontinuation in cystic fibrosis patients homozygous for the F508del CFTR mutation
por: Pohl, Kerstin, et al.
Publicado: (2018)

Elexacaftor/VX-445-mediated CFTR interactome remodeling reveals differential correction driven by mutation-specific translational dynamics
por: Kim, Minsoo, et al.
Publicado: (2023)

Cannot write session to /tmp/vufind_sessions/sess_56dlnveeg13eoe0jn0hrd0l904