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An unexpected effect of TNF-α on F508del-CFTR maturation and function.
F1000Research , 2015 | Pubmed ID: 26594334
The suppression of premature termination codons and the repair of splicing mutations in CFTR.
Current opinion in pharmacology 06, 2017 | Pubmed ID: 29128743
Cis variants identified in F508del complex alleles modulate CFTR channel rescue by small molecules.
Human mutation 04, 2018 | Pubmed ID: 29271547
Factors influencing readthrough therapy for frequent cystic fibrosis premature termination codons.
ERJ open research Jan, 2018 | Pubmed ID: 29497617
Might Brushed Nasal Cells Be a Surrogate for CFTR Modulator Clinical Response?
American journal of respiratory and critical care medicine 01, 2019 | Pubmed ID: 30326728
Emerging Therapeutic Approaches for Cystic Fibrosis. From Gene Editing to Personalized Medicine.
Frontiers in pharmacology , 2019 | Pubmed ID: 30873022
Airway surface liquid acidification initiates host defense abnormalities in Cystic Fibrosis.
Scientific reports 04, 2019 | Pubmed ID: 31019198
Cystic Fibrosis Diagnosis in Newborns, Children, and Adults.
Seminars in respiratory and critical care medicine 12, 2019 | Pubmed ID: 31679154
Author Correction: Airway surface liquid acidification initiates host defense abnormalities in Cystic Fibrosis.
Scientific reports Nov, 2019 | Pubmed ID: 31754179
Urinary Exosomes of Patients with Cystic Fibrosis Unravel CFTR-Related Renal Disease.
International journal of molecular sciences Sep, 2020 | Pubmed ID: 32927759
Modulators of CFTR. Updates on clinical development and future directions.
European journal of medicinal chemistry Mar, 2021 | Pubmed ID: 33524685
New insights into structure and function of bis-phosphinic acid derivatives and implications for CFTR modulation.
Scientific reports 03, 2021 | Pubmed ID: 33767236
Sweat Chloride Testing and Nasal Potential Difference (NPD) Are Primary Outcome Parameters in Treatment with Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators.
Journal of personalized medicine Jul, 2021 | Pubmed ID: 34442373
Author Correction: New insights into structure and function of bis-phosphinic acid derivatives and implications for CFTR modulation.
Scientific reports Sep, 2021 | Pubmed ID: 34526640
Pharmacological chaperones improve intra-domain stability and inter-domain assembly via distinct binding sites to rescue misfolded CFTR.
Cellular and molecular life sciences : CMLS Dec, 2021 | Pubmed ID: 34714360
Correlating genotype with phenotype using CFTR-mediated whole-cell Cl currents in human nasal epithelial cells.
The Journal of physiology Mar, 2022 | Pubmed ID: 34761808
Systemic bis-phosphinic acid derivative restores chloride transport in Cystic Fibrosis mice.
Scientific reports Apr, 2022 | Pubmed ID: 35413967
Institut Necker Enfants Malades
Université de paris
Assistance Publique Hôpitaux de Paris
Université de Paris
Mairead Kelly1,2,
Elise Dreano1,2,
Aurelie Hatton1,2,
Agathe Lepissier1,2,
Anita Golec1,2,
Isabelle Sermet-Gaudelus1,2,3,
Iwona Pranke1,2,3
1, Institut Necker Enfants Malades,
2, Université de Paris,
3Centre de Référence Maladies Rares Mucoviscidose et Maladies apparentées, Assistance Publique Hôpitaux de Paris
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