Research Information System
Acta Pharmaceutica Sciencia, vol.63, no.2, pp.249-270, 2025 (Scopus)
Huntington’s disease is a fatal disease that occurs when the number of CAG trinu-cleotides in the IT-15 gene repeats itself more than 35 times. In this study, modified chitosan polymers were synthesized and characterized. siRNA loaded nano-particles 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 siRNA-loaded 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.