Cargando…

A Precision Medicine Approach to Optimize Modulator Therapy for Rare CFTR Folding Mutants

Trikafta, a triple-combination drug, consisting of folding correctors VX-661 (tezacaftor), VX-445 (elexacaftor) and the gating potentiator VX-770 (ivacaftor) provided unprecedented clinical benefits for patients with the most common cystic fibrosis (CF) mutation, F508del. Trikafta indications were r...

Descripción completa

Detalles Bibliográficos
Autores principales:	Veit, Guido, Velkov, Tony, Xu, Haijin, Vadeboncoeur, Nathalie, Bilodeau, Lara, Matouk, Elias, Lukacs, Gergely L.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8307171/ https://www.ncbi.nlm.nih.gov/pubmed/34357110 http://dx.doi.org/10.3390/jpm11070643

Ejemplares similares

Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination
por: Veit, Guido, et al.
Publicado: (2020)

Folding correctors can restore CFTR posttranslational folding landscape by allosteric domain–domain coupling
por: Soya, Naoto, et al.
Publicado: (2023)

Chaperones rescue the energetic landscape of mutant CFTR at single molecule and in cell
por: Bagdany, Miklos, et al.
Publicado: (2017)

N-glycans are direct determinants of CFTR folding and stability in secretory and endocytic membrane traffic
por: Glozman, Rina, et al.
Publicado: (2009)

Fixing cystic fibrosis by correcting CFTR domain assembly
por: Okiyoneda, Tsukasa, et al.
Publicado: (2012)

Proinflammatory cytokine secretion is suppressed by TMEM16A or CFTR channel activity in human cystic fibrosis bronchial epithelia
por: Veit, Guido, et al.
Publicado: (2012)

Structure-guided combination therapy to potently improve the function of mutant CFTRs
por: Veit, Guido, et al.
Publicado: (2018)

Folding–function relationship of the most common cystic fibrosis–causing CFTR conductance mutants
por: van Willigen, Marcel, et al.
Publicado: (2019)

Mechanisms of CFTR Folding at the Endoplasmic Reticulum
por: Kim, Soo Jung, et al.
Publicado: (2012)

The Primary Folding Defect and Rescue of ΔF508 CFTR Emerge during Translation of the Mutant Domain
por: Hoelen, Hanneke, et al.
Publicado: (2010)

Ribosomal Stalk Protein Silencing Partially Corrects the ΔF508-CFTR Functional Expression Defect
por: Veit, Guido, et al.
Publicado: (2016)

Selective Binding of HSC70 and its Co-Chaperones to Structural Hotspots on CFTR
por: Baaklini, Imad, et al.
Publicado: (2020)

Phosphorylation-dependent modulation of CFTR macromolecular signalling complex activity by cigarette smoke condensate in airway epithelia
por: Schnúr, Andrea, et al.
Publicado: (2019)

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

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

Biophysical Characterisation of Calumenin as a Charged F508del-CFTR Folding Modulator
por: Tripathi, Rashmi, et al.
Publicado: (2014)

Quality control for unfolded proteins at the plasma membrane
por: Apaja, Pirjo M., et al.
Publicado: (2010)

Airway Epithelial Inflammation In Vitro Augments the Rescue of Mutant CFTR by Current CFTR Modulator Therapies
por: Gentzsch, Martina, et al.
Publicado: (2021)

Repairing folding-defective α-sarcoglycan mutants by CFTR correctors, a potential therapy for limb-girdle muscular dystrophy 2D
por: Carotti, Marcello, et al.
Publicado: (2018)

Ins and outs of AlphaFold2 transmembrane protein structure predictions
por: Hegedűs, Tamás, et al.
Publicado: (2022)

Rescue of Rare CFTR Trafficking Mutants Highlights a Structural Location-Dependent Pattern for Correction
por: Zacarias, Sónia, et al.
Publicado: (2023)

CFTR: the nucleotide binding folds regulate the accessibility and stability of the activated state
Publicado: (1996)

Nanomechanics combined with HDX reveals allosteric drug binding sites of CFTR NBD1
por: Padányi, Rita, et al.
Publicado: (2022)

CFTR trafficking mutations disrupt cotranslational protein folding by targeting biosynthetic intermediates
por: Shishido, Hideki, et al.
Publicado: (2020)

Towards next generation therapies for cystic fibrosis: Folding, function and pharmacology of CFTR()
por: Bose, Samuel J., et al.
Publicado: (2020)

Misfolding diverts CFTR from recycling to degradation: quality control at early endosomes
por: Sharma, Manu, et al.
Publicado: (2004)

Bithiazole Correctors Rescue CFTR Mutants by Two Different Mechanisms
por: Loo, Tip W., et al.
Publicado: (2013)

Evaluation of Fused Pyrrolothiazole Systems as Correctors of Mutant CFTR Protein
por: Spanò, Virginia, et al.
Publicado: (2021)

Production of CFTR Mutant Gene Model by Homologous Recombination System
por: Rezaee, Hanieh, et al.
Publicado: (2022)

CFTR Modulators: Shedding Light on Precision Medicine for Cystic Fibrosis
por: Lopes-Pacheco, Miquéias
Publicado: (2016)

Precision Medicine Based on CFTR Genotype for People with Cystic Fibrosis
por: Haq, Iram, et al.
Publicado: (2022)

Role of Hsc70 binding cycle in CFTR folding and endoplasmic reticulum–associated degradation
por: Matsumura, Yoshihiro, et al.
Publicado: (2011)

Folding Status Is Determinant over Traffic-Competence in Defining CFTR Interactors in the Endoplasmic Reticulum
por: Santos, João D., et al.
Publicado: (2019)

Spatial covariance analysis reveals the residue-by-residue thermodynamic contribution of variation to the CFTR fold
por: Anglès, Frédéric, et al.
Publicado: (2022)

ABC-transporter CFTR folds with high fidelity through a modular, stepwise pathway
por: Im, Jisu, et al.
Publicado: (2023)

Epithelial Anion Transport as Modulator of Chemokine Signaling
por: Schnúr, Andrea, et al.
Publicado: (2016)

From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations
por: Veit, Gudio, et al.
Publicado: (2016)

Correcting the Cystic Fibrosis Disease Mutant, A455E CFTR
por: Cebotaru, Liudmila, et al.
Publicado: (2014)

Mutant CFTR Drives TWIST1 mediated epithelial–mesenchymal transition
por: Quaresma, Margarida C., et al.
Publicado: (2020)

Redefining Hypo- and Hyper-Responding Phenotypes of CFTR Mutants for Understanding and Therapy
por: Hillenaar, Tamara, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_bt19v2dk9h1prb6t2a7s1mbruh