Publications by authors named "Sebastien Terreau"
Highly branched poly β-amino ester/CpG-depleted plasmid nanoparticles for non-viral gene therapy in lung cystic fibrosis disease.
Mol Ther Methods Clin Dev
September 2024
Lung cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF transmembrane conductance regulator () gene, leading to a dysfunctional CFTR protein. Gene therapy offers promise for the treatment of lung CF. However, the development and clinical application of CF gene therapy have long been hampered by the absence of safe and highly efficient delivery vectors.
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- The study explores a new method for concentrating highly branched poly(β-amino ester) (HPAEs)/DNA nanoparticles for gene therapy aimed at treating cystic fibrosis, addressing challenges related to the high formulation concentrations required for clinical applications.
- Researchers optimized a formulation using various buffers and achieved concentration through ultrafiltration, which significantly outperformed lyophilization by providing a 24-fold increase.
- The concentrated formulation was effective in restoring CFTR protein production in lung epithelial cells, demonstrating better results than existing transfection reagents, highlighting its potential for future preclinical testing and clinical use.
Microglial cells play important roles in neurodegenerative diseases including peroxisomal leukodystrophies. The BV-2 murine immortalized cells are widely used in the context of neurodegenerative researches. It is therefore important to establish the expression pattern of peroxisomal proteins by flow cytometry in these cells.
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