Development and in vitro characterization studies of novel chitosan nanoparticles for the treatment of Huntington’s disease


Çağlar E. Ş., Eker Fidan E. B., GÖK M. K., Üstündağ Okur N., Pabuccuoğlu S. K., CEVHER E.

Acta Pharmaceutica Sciencia, cilt.63, sa.2, ss.249-270, 2025 (Scopus) identifier

  • Yayın Türü: Makale / Tam Makale
  • Cilt numarası: 63 Sayı: 2
  • Basım Tarihi: 2025
  • Doi Numarası: 10.23893/1307-2080.aps6316
  • Dergi Adı: Acta Pharmaceutica Sciencia
  • Derginin Tarandığı İndeksler: Scopus, EMBASE
  • Sayfa Sayıları: ss.249-270
  • Anahtar Kelimeler: chitosan, Huntington’s disease, nanoparticle, siRNA
  • İstanbul Üniversitesi Adresli: Evet

Özet

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.