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ACTA Pharmaceutica Sciencia
2025 , Vol 63 , Num 2
Development and in vitro characterization studies of novel chitosan nanoparticles for the treatment of Huntington's disease
1 University of Health Sciences, Faculty of Pharmacy, Department of Pharmaceutical Biotechnology, Istanbul, Türkiye2 Istanbul University-Cerrahpasa, Faculty of Engineering, Department of Chemical Engineering, Istanbul, Türkiye
3 University of Health Sciences, Faculty of Pharmacy, Department of Pharmaceutical Technology, Istanbul, Türkiye
4 Istanbul University, Faculty of Pharmacy, Department of Pharmaceutical Technology, Istanbul, Türkiye
DOI : 10.23893/1307-2080.APS6316 Viewed : 3483 - Downloaded : 1580 Huntington"s disease is a fatal disease that occurs when the number of CAG trinucleotides in the IT-15 gene repeats itself more than 35 times. In this study, modified chitosan polymers were synthesized and characterized. siRNA loaded nanoparticles were prepared and characterized. The results showed that the modified chitosan was successfully synthesized and targeted. As a result of the characterization studies, the particle size, polydispersity index and zeta potential of the siRNAloaded nanoparticle were found to be 99.0 ± 5.1 nm, 0.3190 ± 0.004, and 14.9 ± 3.04 mV, respectively. Lyophilization was successfully achieved with a trehalose rate of 20%. As a result of 12 months of stability, the particle size of the formulations was found to be the highest 273.766 ± 8.957 nm, the highest polydispersity index was 0.324 ± 0.016, and the highest zeta potential was 45.35 ± 1.79 mV. In agarose gel electrophoresis studies, the siRNA:modified chitosan ratio was found to be 5:1. siRNA-loaded nanoparticles maintain the integrity of siRNA for 24h. Keywords : Huntington's disease, siRNA, chitosan, nanoparticle