Gene therapy potential for genetic disorders of surfactant dysfunction

AL Cooney, JA Wambach, PL Sinn… - Frontiers in genome …, 2022 - frontiersin.org
Pulmonary surfactant is critically important to prevent atelectasis by lowering the surface
tension of the alveolar lining liquid. While respiratory distress syndrome (RDS) is common in …

Pulmonary surfactant: a unique biomaterial with life-saving therapeutic applications

B Pioselli, F Salomone, G Mazzola… - Current Medicinal …, 2022 - ingentaconnect.com
Pulmonary surfactant is a complex lipoprotein mixture secreted into the alveolar lumen by
type 2 pneumocytes, which is composed by tens of different lipids (approximately 90% of its …

Mass spectrometry and machine learning reveal determinants of client recognition by antiamyloid chaperones

N Österlund, T Vosselman, A Leppert… - Molecular & Cellular …, 2022 - ASBMB
The assembly of proteins and peptides into amyloid fibrils is causally linked to serious
disorders such as Alzheimer's disease. Multiple proteins have been shown to prevent …

Gene therapeutics for surfactant dysfunction disorders: targeting the alveolar type 2 epithelial cell

S Sitaraman, KD Alysandratos, JA Wambach… - Human Gene …, 2022 - liebertpub.com
Genetic disorders of surfactant dysfunction result in significant morbidity and mortality,
among infants, children, and adults. Available medical interventions are limited, nonspecific …

Immunophenotyping of acute inflammatory exacerbations of lung injury driven by mutant surfactant protein-C: a role for inflammatory eosinophils

J Nguyen, BS Armstrong, S Cowman… - Frontiers in …, 2022 - frontiersin.org
Acute inflammatory exacerbations (AIEs) represent immune-driven deteriorations of many
chronic lung conditions, including COPD, asthma, and pulmonary fibrosis (PF). The first line …

Etiologic Profile of older children with diffuse radiological changes in Eastern China

X Xu, L Liu, X Xu, Q Ma, L Teng, H Zhou, L Yang… - Frontiers in …, 2022 - frontiersin.org
Objective: To analyze the etiology of chest diffuse radiological changes (DRC) in children
older than 2 years. Methods A retrospective study was conducted on a primary cohort of …

Association analysis of the surfactant protein-C gene to childhood asthma

M Nefzi, I Wahabi, S Hadj Fredj, R Othmani… - Journal of …, 2022 - Taylor & Francis
Objectives This study aims to describe the molecular variability in the SFTPC gene in a
childhood chronic respiratory disease, asthma, in the Tunisian population and to identify the …

Compositions and methods for in utero gene editing for monogenic lung disease

WH Peranteau, EE Morrisey, MF Beers… - US Patent App. 17 …, 2022 - Google Patents
In accordance with the present invention, compositions and a CRISPR-Cas system-
mediated genome editing method are disclosed. An exemplary method comprises …