Cargando…

Emerging Therapeutic Approaches for Cystic Fibrosis. From Gene Editing to Personalized Medicine

An improved understanding of the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) protein structure and the consequences of CFTR gene mutations have allowed the development of novel therapies targeting specific defects underlying CF. Some strategies are mutation specific and have alre...

Descripción completa

Detalles Bibliográficos
Autores principales:	Pranke, Iwona, Golec, Anita, Hinzpeter, Alexandre, Edelman, Aleksander, Sermet-Gaudelus, Isabelle
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2019
Materias:	Pharmacology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6400831/ https://www.ncbi.nlm.nih.gov/pubmed/30873022 http://dx.doi.org/10.3389/fphar.2019.00121

Ejemplares similares

Urinary Exosomes of Patients with Cystic Fibrosis Unravel CFTR-Related Renal Disease
por: Gauthier, Sebastien, et al.
Publicado: (2020)

Factors influencing readthrough therapy for frequent cystic fibrosis premature termination codons
por: Pranke, Iwona, et al.
Publicado: (2018)

Airway surface liquid acidification initiates host defense abnormalities in Cystic Fibrosis
por: Simonin, Juliette, et al.
Publicado: (2019)

Acting on the CFTR Membrane-Spanning Domains Interface Rescues Some Misfolded Mutants
por: Baatallah, Nesrine, et al.
Publicado: (2022)

Differential CFTR-Interactome Proximity Labeling Procedures Identify Enrichment in Multiple SLC Transporters
por: Chevalier, Benoît, et al.
Publicado: (2022)

Author Correction: Airway surface liquid acidification initiates host defense abnormalities in Cystic Fibrosis
por: Simonin, Juliette, et al.
Publicado: (2019)

An unexpected effect of TNF-α on F508del-CFTR maturation and function
por: Bitam, Sara, et al.
Publicado: (2015)

Sweat Chloride Testing and Nasal Potential Difference (NPD) Are Primary Outcome Parameters in Treatment with Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators
por: Sermet-Gaudelus, Isabelle, et al.
Publicado: (2021)

Systemic bis-phosphinic acid derivative restores chloride transport in Cystic Fibrosis mice
por: da Cunha, Mélanie Faria, et al.
Publicado: (2022)

Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators
por: Pranke, Iwona M., et al.
Publicado: (2017)

Insights Into Patient Variability During Ivacaftor-Lumacaftor Therapy in Cystic Fibrosis
por: Hanafin, Patrick O., et al.
Publicado: (2021)

The CF-CIRC study: a French collaborative study to assess the accuracy of Cystic Fibrosis diagnosis in neonatal screening
por: Sermet-Gaudelus, Isabelle, et al.
Publicado: (2006)

Personalized or Precision Medicine? The Example of Cystic Fibrosis
por: Marson, Fernando A. L., et al.
Publicado: (2017)

A Novel Lipidomic Strategy Reveals Plasma Phospholipid Signatures Associated with Respiratory Disease Severity in Cystic Fibrosis Patients
por: Guerrera, Ida Chiara, et al.
Publicado: (2009)

Achromobacter xylosoxidans airway infection is associated with lung disease severity in children with cystic fibrosis
por: Marsac, Charlotte, et al.
Publicado: (2021)

On the Corner of Models and Cure: Gene Editing in Cystic Fibrosis
por: Ensinck, Marjolein, et al.
Publicado: (2021)

In vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis: a pilot study
por: Sermet-Gaudelus, Isabelle, et al.
Publicado: (2007)

Editorial: Emerging Therapeutic Approaches for Cystic Fibrosis
por: Lopes-Pacheco, Miquéias, et al.
Publicado: (2019)

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

Therapeutic Approaches for Patients with Cystic Fibrosis Not Eligible for Current CFTR Modulators
por: Fajac, Isabelle, et al.
Publicado: (2021)

Impact of COVID-19 on people with cystic fibrosis
por: Colombo, Carla, et al.
Publicado: (2020)

Emerging microRNA Therapeutic Approaches for Cystic Fibrosis
por: Bardin, Pauline, et al.
Publicado: (2018)

Pseudomonas aeruginosa LasB protease impairs innate immunity in mice and humans by targeting a lung epithelial cystic fibrosis transmembrane regulator–IL-6–antimicrobial–repair pathway
por: Saint-Criq, Vinciane, et al.
Publicado: (2018)

Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized Medicine
por: Awatade, Nikhil T., et al.
Publicado: (2018)

New insights into structure and function of bis-phosphinic acid derivatives and implications for CFTR modulation
por: Bitam, Sara, et al.
Publicado: (2021)

Author Correction: New insights into structure and function of bis-phosphinic acid derivatives and implications for CFTR modulation
por: Bitam, Sara, et al.
Publicado: (2021)

Characterization of Nasal Potential Difference in cftr Knockout and F508del-CFTR Mice
por: Saussereau, Emilie Lyne, et al.
Publicado: (2013)

Elexacaftor/Tezacaftor/Ivacaftor Disrupts Respiratory Tract Development in a Murine Fetal Lung Explant Model
por: Lhuillier, Mickaël, et al.
Publicado: (2022)

Isolation, cultivation, and application of primary respiratory epithelial cells obtained by nasal brushing, polyp samples, or lung explants
por: Golec, Anita, et al.
Publicado: (2022)

Moving the Dial on Airway Inflammation in Response to Trikafta in Adolescents with Cystic Fibrosis
por: Lepissier, Agathe, et al.
Publicado: (2023)

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

Mycobacterium abscessus and Children with Cystic Fibrosis
por: Sermet-Gaudelus, Isabelle, et al.
Publicado: (2003)

Role of the SLC26A9 Chloride Channel as Disease Modifier and Potential Therapeutic Target in Cystic Fibrosis
por: Balázs, Anita, et al.
Publicado: (2018)

β‐adrenergic sweat test in children with inconclusive cystic fibrosis diagnosis: Do we need new reference ranges?
por: Zampoli, Marco, et al.
Publicado: (2022)

CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine
por: Lopes-Pacheco, Miquéias
Publicado: (2020)

Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial
por: Quittner, Alexandra, et al.
Publicado: (2015)

Correction to: In vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis: a pilot study
por: Sermet-Gaudelus, I., et al.
Publicado: (2018)

Innovative Therapeutic Strategies for Cystic Fibrosis: Moving Forward to CRISPR Technique
por: Marangi, Michele, et al.
Publicado: (2018)

Interest of colchicine for the treatment of cystic fibrosis patients. Preliminary report.
por: Sermet-Gaudelus, I, et al.
Publicado: (1999)

Proteomic profiling of sweat in patients with cystic fibrosis provides new insights into epidermal homoeostasis
por: Cornet, Matthieu, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_jrq83hjahmfej7up6p3m5tsuom