Cargando…

Integrity and Stability of PTC Bearing CFTR mRNA and Relevance to Future Modulator Therapies in Cystic Fibrosis

Major advances have recently been made in the development and application of CFTR (cystic fibrosis transmembrane conductance regulator) mutation class-specific modulator therapies, but to date, there are no approved modulators for Class I mutations, i.e., those introducing a premature termination co...

Descripción completa

Detalles Bibliográficos
Autores principales:	Clarke, Luka A., Luz, Vanessa C. C., Targowski, Szymon, Ramalho, Sofia S., Farinha, Carlos M., Amaral, Margarida D.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8621375/ https://www.ncbi.nlm.nih.gov/pubmed/34828417 http://dx.doi.org/10.3390/genes12111810

Ejemplares similares

Rare Trafficking CFTR Mutations Involve Distinct Cellular Retention Machineries and Require Different Rescuing Strategies
por: Ramalho, Sofia S., et al.
Publicado: (2021)

Personalized medicine: Function of CFTR variant p.Arg334Trp is rescued by currently available CFTR modulators
por: Railean, Violeta, et al.
Publicado: (2023)

CFTR mutations altering CFTR fragmentation
por: Tosoni, Kendra, et al.
Publicado: (2012)

Cysteamine re-establishes the clearance of Pseudomonas aeruginosa by macrophages bearing the cystic fibrosis-relevant F508del-CFTR mutation
por: Ferrari, Eleonora, et al.
Publicado: (2017)

Rescue of Mutant CFTR Trafficking Defect by the Investigational Compound MCG1516A
por: Lopes-Pacheco, Miquéias, et al.
Publicado: (2022)

Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis
por: Awatade, Nikhil T., et al.
Publicado: (2014)

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

LMTK2-mediated Phosphorylation Regulates CFTR Endocytosis in Human Airway Epithelial Cells
por: Luz, Simão, et al.
Publicado: (2014)

Enhanced Expression of Human Epididymis Protein 4 (HE4) Reflecting Pro-Inflammatory Status Is Regulated by CFTR in Cystic Fibrosis Bronchial Epithelial Cells
por: Bene, Zsolt, et al.
Publicado: (2021)

Synergy in Cystic Fibrosis Therapies: Targeting SLC26A9
por: Pinto, Madalena C., et al.
Publicado: (2021)

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

Full Rescue of F508del-CFTR Processing and Function by CFTR Modulators Can Be Achieved by Removal of Two Regulatory Regions
por: Uliyakina, Inna, et al.
Publicado: (2020)

Changes in transcriptome of native nasal epithelium expressing F508del-CFTR and intersecting data from comparable studies
por: Clarke, Luka A, et al.
Publicado: (2013)

Absence of EPAC1 Signaling to Stabilize CFTR in Intestinal Organoids
por: Ferreira, João F., et al.
Publicado: (2022)

KLF4 Acts as a wt-CFTR Suppressor through an AKT-Mediated Pathway
por: Sousa, Luis, et al.
Publicado: (2020)

CFTR, Cell Junctions and the Cytoskeleton
por: Pankonien, Ines, et al.
Publicado: (2022)

Cystic Fibrosis, CFTR, and Colorectal Cancer
por: Scott, Patricia, et al.
Publicado: (2020)

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

Measurements of CFTR-Mediated Cl(−) Secretion in Human Rectal Biopsies Constitute a Robust Biomarker for Cystic Fibrosis Diagnosis and Prognosis
por: Sousa, Marisa, et al.
Publicado: (2012)

Mechanical Properties of Human Bronchial Epithelial Cells Expressing Wt- and Mutant CFTR
por: Carapeto, Ana P., et al.
Publicado: (2020)

CFTR and Ca(2+) Signaling in Cystic Fibrosis
por: Antigny, Fabrice, et al.
Publicado: (2011)

Strategies to circumvent the CFTR defect in cystic fibrosis
por: Becq, Frédéric, et al.
Publicado: (2013)

Not All Is CFTR – Neutrophils and Cholesterol in Cystic Fibrosis
por: Castellani, Stefano, et al.
Publicado: (2017)

Cystic Fibrosis and Oxidative Stress: The Role of CFTR
por: Moliteo, Evelina, et al.
Publicado: (2022)

CFTR modulators: transformative therapies for cystic fibrosis
por: Dwight, Mary, et al.
Publicado: (2021)

Restoration of CFTR function in patients with cystic fibrosis carrying the F508del-CFTR mutation
por: Stefano, Daniela De, et al.
Publicado: (2014)

Molecular Chaperones as Targets to Circumvent the CFTR Defect in Cystic Fibrosis
por: Chanoux, Rebecca A., et al.
Publicado: (2012)

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)

The cystic fibrosis transmembrane conductance regulator (CFTR) and its stability
por: Meng, Xin, et al.
Publicado: (2016)

What Role Does CFTR Play in Development, Differentiation, Regeneration and Cancer?
por: Amaral, Margarida D., et al.
Publicado: (2020)

The K(+) Channel Opener 1-EBIO Potentiates Residual Function of Mutant CFTR in Rectal Biopsies from Cystic Fibrosis Patients
por: Roth, Eva K., et al.
Publicado: (2011)

Inflammation and CFTR: might neutrophils be the key in cystic fibrosis?
por: Witko-Sarsat, V, et al.
Publicado: (1999)

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

Targeted therapies to improve CFTR function in cystic fibrosis
por: Brodlie, Malcolm, et al.
Publicado: (2015)

Lived experiences of individuals with cystic fibrosis on CFTR-modulators
por: Page, Annelise, et al.
Publicado: (2022)

The Effect of CFTR Modulators on Airway Infection in Cystic Fibrosis
por: Harvey, Caitlyn, et al.
Publicado: (2022)

Nutritional impact of CFTR modulators in children with cystic fibrosis
por: Gaschignard, Margaux, et al.
Publicado: (2023)

Cross‐talk between CFTR and sphingolipids in cystic fibrosis
por: Dobi, Dorina, et al.
Publicado: (2023)

Mutation-class dependent signatures outweigh disease-associated processes in cystic fibrosis cells
por: Santos, Lúcia, et al.
Publicado: (2023)

Capsaicin-Loaded Chitosan Nanocapsules for wtCFTR-mRNA Delivery to a Cystic Fibrosis Cell Line
por: Kolonko, A. Katharina, et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_ifu71gnn9hdionp19it2tll2mm