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- W4308383492 endingPage "114617" @default.
- W4308383492 startingPage "114617" @default.
- W4308383492 abstract "Cyclodextrin (CD), a cyclic oligosaccharide, is a pharmaceutical additive that improves the solubility of hydrophobic compounds. Recent research has focused on the potential active pharmaceutical abilities of CD. Lysosomal storage diseases are inherited metabolic diseases characterized by lysosomal dysfunction and abnormal lipid storage. Niemann-Pick disease type C (NPC) is caused by mutations in cholesterol transporter genes (NPC1, NPC2) and is characterized by cholesterol accumulation in lysosomes. A biocompatible cholesterol solubilizer 2-hydroxypropyl-β-cyclodextrin (HP-β-CD) was recently used in NPC patients for compassionate use and in clinical trials. HP-β-CD is an attractive drug candidate for NPC; however, its adverse effects, such as ototoxicity, should be solved. In this review, we discuss the current use of HP-β-CD in basic and clinical research and discuss alternative CD derivatives that may outperform HP-β-CD, which should be considered for clinical use. The potential of CD therapy for the treatment of other lysosomal storage diseases is also discussed." @default.
- W4308383492 created "2022-11-11" @default.
- W4308383492 creator A5025216257 @default.
- W4308383492 creator A5027112145 @default.
- W4308383492 creator A5035850424 @default.
- W4308383492 date "2022-12-01" @default.
- W4308383492 modified "2023-09-26" @default.
- W4308383492 title "Cyclodextrins applied to the treatment of lysosomal storage disorders" @default.
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