Feasibility study of cyclodextrins as active pharmaceutical ingredients for the treatment of GM1-gangliosidosis

作     者：Yuki Maeda Keiichi Motoyama Taishi Higashi Yuka Horikoshi Toru Takeo Naomi Nakagata Yuki Kurauchi Hiroshi Katsuki Yuki Kondo Yoichi Ishitsuka Tetsumi Irie Takumi Erad Hidetoshi Arima 

作者机构：Graduate School of Pharmaceutical SciencesKumamoto University5-1 Oe-honmachiChuo-kuKumamotoJapan Program for Leading Graduate Schools“HIGO(Health life science:Interdisciplinary and Glocal Oriented)Program”Kumamoto University5-1 Oe-honmachiChuo-kuKumamotoJapan Center for Animal Resources and DevelopmentKumamoto University2-2-1 HonjoChou-kuKumamotoJapan Institute of Molecular Embryology and GeneticsKumamoto University2-2-1 HonjoChou-kuKumamotoJapan 

出 版 物：《Asian Journal of Pharmaceutical Sciences》 (亚洲药物制剂科学（英文）)

年 卷 期：2016年第11卷第1期

页      面：183-184页

学科分类：1007[医学-药学(可授医学、理学学位)] 1006[医学-中西医结合] 100706[医学-药理学] 100602[医学-中西医结合临床] 10[医学] 

基　　金：the financial supports received from Health and Labor Sciences Research Grant in Japan (201406032A) HIGO Program Research Funding Project (Fiscal Year 2014) 

主　　题：Cyclodextrins GM1-gangliosidosis Fibroblasts,Lysosomes 

摘      要：GM1-gangliosidosis is a rare lysosomal storage disorder characterized clinically by a wide range of variable neurovisceral,ophthalmological and dysmorphic features. Without enough functionalβ-galactosidase, GM1-gangliosides cannot be degraded in lysosomes, and accumulate to toxic levels in many tissues and organs, particularly in the brain. In spite of several approaches for the treatment of GM1-gangliosidosis.