Marie E. Egan
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View article: Chronic pulmonary LPS exposure in cystic fibrosis drives maladaptive pro-inflammatory hematopoietic stem and progenitor cells
Chronic pulmonary LPS exposure in cystic fibrosis drives maladaptive pro-inflammatory hematopoietic stem and progenitor cells Open
Background: Persistent lung inflammation characterizes cystic fibrosis (CF), mainly driven by myeloid cells (neutrophils and monocytes) that can contribute to tissue damage and disease progression. Hematopoietic stem and progenitor cells (…
View article: Systemic in utero gene editing as a treatment for cystic fibrosis
Systemic in utero gene editing as a treatment for cystic fibrosis Open
In utero gene editing has the potential to modify disease-causing genes in multiple developing tissues before birth, possibly allowing for normal organ development, disease improvement, and conceivably, cure. In cystic fibrosis (CF), a dis…
View article: Sputum Induction in Pediatric Lung Disease: Are We There Yet?
Sputum Induction in Pediatric Lung Disease: Are We There Yet? Open
Sputum induction (SI) is a non-invasive technique to obtain lower airway samples from people who do not spontaneously produce sputum. Since its introduction in 1958, its use has significantly increased for various respiratory conditions, e…
View article: Systemic in utero gene editing as a treatment for cystic fibrosis
Systemic in utero gene editing as a treatment for cystic fibrosis Open
In utero gene editing has the potential to modify disease causing genes in multiple developing tissues before birth, possibly allowing for normal organ development, disease improvement, and conceivably, cure. In cystic fibrosis (CF), a dis…
View article: CHRONIC LUNG INFLAMMATION LEADS TO MYELOID SKEWING OF HEMATOPOIETIC STEM CELLS IN A CYSTIC FIBROSIS MOUSE MODEL
CHRONIC LUNG INFLAMMATION LEADS TO MYELOID SKEWING OF HEMATOPOIETIC STEM CELLS IN A CYSTIC FIBROSIS MOUSE MODEL Open
Persistent lung inflammation is a hallmark of Cystic fibrosis (CF) lung disease. Inflammation can lead to functional decline in hematopoietic stem cell (HSCs), tipping the balance towards myelopoiesis and contributing to chronic inflammati…
View article: Localization and function of humanized F508del-CFTR in mouse intestine following activation of serum glucocorticoid kinase 1 and Trikafta
Localization and function of humanized F508del-CFTR in mouse intestine following activation of serum glucocorticoid kinase 1 and Trikafta Open
The CFTR modulator Trikafta has markedly improved lung disease for Cystic Fibrosis (CF) patients carrying the common delta F508 (F508del-CFTR) CFTR mutation. F508del-CFTR results in an apical trafficking defect and loss of function in CFTR…
View article: The Cystic Fibrosis Transmembrane Regulator Controls Tolerogenic Responses to Food Allergens in Mice and Humans
The Cystic Fibrosis Transmembrane Regulator Controls Tolerogenic Responses to Food Allergens in Mice and Humans Open
IgE antibodies against the allergen Ara-h2 can cause life-threatening anaphylaxis upon exposure to peanuts. Desensitization strategies aim at inducing IgG responses against Ara-h2 which may compete with anaphylactogenic IgE. Here we assess…
View article: In vivo correction of cystic fibrosis mediated by PNA nanoparticles
In vivo correction of cystic fibrosis mediated by PNA nanoparticles Open
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. We sought to correct the multiple organ dysfunction of the F508del CF-causing mutation using systemic delivery of peptide nucleic acid g…
View article: Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells
Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells Open
Background Advances in Molecular Therapy have made gene editing through systemic or topical administration of reagents a feasible strategy to treat genetic diseases in a rational manner. Encapsulation of therapeutic agents in nanoparticles…
View article: <i>In vivo</i> correction of cystic fibrosis mediated by PNA nanoparticles
<i>In vivo</i> correction of cystic fibrosis mediated by PNA nanoparticles Open
Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. We sought to correct the multiple organ dysfunction of the F508del CF-causing mutation using systemic delivery of peptide n…
View article: Understanding the Host in the Management of Pneumonia. An Official American Thoracic Society Workshop Report
Understanding the Host in the Management of Pneumonia. An Official American Thoracic Society Workshop Report Open
Pneumonia causes a significant burden of disease worldwide. Although all populations are at risk of pneumonia, those at extremes of age and those with immunosuppressive disorders, underlying respiratory disease, and critical illness are pa…
View article: SPLUNC1: a novel marker of cystic fibrosis exacerbations
SPLUNC1: a novel marker of cystic fibrosis exacerbations Open
Background Acute pulmonary exacerbations (AE) are episodes of clinical worsening in cystic fibrosis (CF), often precipitated by infection. Timely detection is critical to minimise morbidity and lung function declines associated with acute …
View article: Emerging technologies for cystic fibrosis transmembrane conductance regulator restoration in all people with CF
Emerging technologies for cystic fibrosis transmembrane conductance regulator restoration in all people with CF Open
Although effective cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy has the potential to change the lives of many patients with cystic fibrosis (CF), it is unlikely that these drugs will be a game changing thera…