Posted by A β-CDP (CycloLab) was used for the experiments. The purity of the β-CDP was >95 %. The β-CDP contained <1 % of uncrosslinked β-CD.
Table 1. Characterization of β-CDP.
| Mw (kDa)a | Mean diameter (nm)b | Polydispersity indexb | β-CD content (%)c | No. of β-CD | Hemolitic activity (mg/mL)d |
|---|---|---|---|---|---|
| 114.8 ± 1.8 | 23.3 | 0.34 | 65.0 | 75 | 80.7 |
- a Measured by static light scattering using Zetasizer Pro. The specific refractive index increment (dn/dc) was 0.14 mL/g in water.
- b Measured by dynamic light scattering using Zetasizer Pro.
- c Calculated using the peaks of 1H NMR.
- d The concentration of β-CDP to induce 50 % hemolysis of rat erythrocytes.
The β-CD content in β-CDP was calculated from the integrated values of the peaks obtained using 1H NMR suggesting that β-CDP contains approximately 75 β-CD in a molecule with a 65 % weight ratio. The hemolytic activity of β-CDP against rat erythrocytes was evaluated, and the concentration causing 50 % hemolysis was 80.7 mg/mL. In addition, the degradation behavior of β-CDP under physiological conditions was evaluated in terms of molecular weight, particle size, and degradation products after incubation in PBS (pH 7.4) at 37 °C for 24 h. No change was observed.
β-CDP with polymeric structure showed prolonged half-life. It not only reduced cholesterol accumulation more effectively than HP-β-CD but also ameliorate autophagy dysfunction while exhibiting an improved safety profile, particularly with respect to pulmonary toxicity. It also reduced hepatic free cholesterol levels and hepatomegaly in NPC model mice, even at lower doses. These results suggest that β-CDP may have potential as a therapeutic agent for Niemann Pick disease (NPC) and Alzhimer disease (AD).
Yuto Kubohira, Nanami Okano, Toru Taharabaru, Arina Ishimatsu, Takumi Era, Kazunori Yanagihara, Kandai Ishikura, Yoshinori Nakagawa, Taishi Higashi, Keiichi Motoyama (2026) A water-soluble β-cyclodextrin polymer reduces cholesterol accumulation and autophagy dysfunction in vitro and in Niemann-Pick type C disease model mice. Carbohydrate Polymers 374, 124676. https://doi.org/10.1016/j.carbpol.2025.124676.