Fluorescent PLGA from PolySciTech used in development of gene-editing therapy for treatment of cystic fibrosis

 

Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. Gene-editing technology could treat this disease at the source by altering the DNA to provide for a corrected CFTR gene. Recently, researchers at Yale University used PLGA-CY5 (Cat# AV034) from PolySciTech Division of Akina, Inc. (www.polyscitech.com) to create nanoparticles that were fluorescently traceable. They used these for testing a delivery mechanism of peptide nucleic acid gene editing technology as a therapy for CF. This research holds promise to improve treatment of this fatal disease. Read More: Piotrowski-Daspit, A.S., Barone, C., Lin, C.Y., Deng, Y., Wu, D., Binns, T.C., Xu, E., Ricciardi, A.S., Putman, R., Garrison, A. and Nguyen, R., 2022. In vivo correction of cystic fibrosis mediated by PNA nanoparticles. Science Advances, 8(40), p.eabo0522. https://www.science.org/doi/abs/10.1126/sciadv.abo0522

“Abstract: 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 gene editing technology mediated by biocompatible polymeric nanoparticles. We confirmed phenotypic and genotypic modification in vitro in primary nasal epithelial cells from F508del mice grown at air-liquid interface and in vivo in F508del mice following intravenous delivery. In vivo treatment resulted in a partial gain of CFTR function in epithelia as measured by in situ potential differences and Ussing chamber assays and correction of CFTR in both airway and GI tissues with no off-target effects above background. Our studies demonstrate that systemic gene editing is possible, and more specifically that intravenous delivery of PNA NPs designed to correct CF-causing mutations is a viable option to ameliorate CF in multiple affected organs.”