Aim: Aim of the study was the development of ζ potential changing nanoparticles as gene delivery system for the cystic fibrosis transmembrane conductance regulator gene.
Methods: Chitosan and carboxymethyl cellulose were modified with phosphotyrosine, a substrate for the brush border enzyme alkaline phosphatase. With these synthesized derivatives, different nanoparticle formulations, including the cystic fibrosis transmembrane conductance regulator gene were prepared by ionic gelation.
Results: A change from negative to positive ζ potential after enzymatic cleavage could be observed. Transfection studies with HEK-293 and Caco-2 cells showed transfection rates comparable to Lipofectamine 2000. Transfection efficiencies were significantly decreased when phosphate cleavage and thus ζ potential change was inhibited by phosphatase inhibitor.
Conclusion: The developed nanoparticles represent a promising gene delivery system.
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http://dx.doi.org/10.2217/nnm-2017-0115 | DOI Listing |
J Cyst Fibros
January 2025
Department of Microbiology and Immunology, Geisel School of Medicine at Dartmouth, Lebanon, NH, USA; Department of Epidemiology, Geisel School of Medicine at Dartmouth, Lebanon, NH, USA. Electronic address:
Background: Adult people with cystic fibrosis (PwCF) have a higher risk of end-stage kidney disease than the general population. The nature and mechanism of kidney disease in CF are unknown. This study quantifies urinary kidney injury markers and examines the hypothesis that neutrophil activation and lung infection are associated with early kidney injury in CF.
View Article and Find Full Text PDFBiochim Biophys Acta Mol Basis Dis
January 2025
Department of Nephrology, Shanghai East Hospital, Tongji University School of Medicine, Shanghai, China; Department of Medicine, Rhode Island Hospital and Alpert Medical School, Brown University, Providence, RI, USA. Electronic address:
Autosomal dominant polycystic kidney disease (ADPKD) is the fourth leading cause of end-stage renal disease, contributing substantially to patient morbidity, mortality, and healthcare system strain. Emerging research highlights a pivotal role of epigenetics in ADPKD's pathophysiology, where mechanisms like DNA methylation, histone modifications, and non-coding RNA regulation significantly impact disease onset and progression. These epigenetic factors influence gene expression and regulate key processes involved in cyst formation and expansion, fibrosis, and inflammatory infiltration, thus accelerating ADPKD progression.
View Article and Find Full Text PDFBackground: Studies showed significant physical improvement after starting elexacaftor/tezacaftor/ivacaftor (ETI). However, some patients reported new mental health symptoms.
Aim: This study explores the impact of ETI on end-stage cystic fibrosis patients, focusing on mental health.
Sci Rep
January 2025
CEINGE-Biotecnologie avanzate Franco Salvatore, Via G. Salvatore 486, Naples, 80145, Italy.
The development of targeted therapies that correct the effect of mutations in patients with cystic fibrosis (CF) and the relevant heterogeneity of the clinical expression of the disease require biomarkers correlated to the severity of the disease useful for monitoring the therapeutic effects. We applied a targeted metabolomic approach by LC-MS/MS on saliva samples from 70 adult CF patients and 63 age/sex-matched controls to investigate alterations in metabolic pathways related to pancreatic insufficiency (PI), Pseudomonas aeruginosa (PA) colonization, CF liver disease (CFLD), and CF related diabetes (CFRD). Sixty salivary metabolites were differentially expressed, with 11 being less abundant and 49 more abundant in CF patients.
View Article and Find Full Text PDFA 3-year-old girl treated with intravenous chemotherapy for bilateral retinoblastoma (RB) and a standard technique of intravitreal topotecan for vitreous seeds in the left eye developed a conjunctival nodule at the injection site. Ultrasound biomicroscopy showed normal underlying sclera and ciliary body. Fundus examination of the left eye showed partly calcified vitreous seeds.
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