Differential mode of cholesterol inclusion with 2-hydroxypropyl-cyclodextrins increases safety margin in treatment of Niemann-Pick disease type C.
Yusei YamadaYoichi IshitsukaYuki KondoShuichi NakaharaAsami NishiyamaNobuyuki MikodaNaomi NakagataKeiichi MotoyamaTaishi HigashiHidetoshi ArimaShunsuke KameiTsuyoshi ShutoHirofumi KaiYuji HayashinoMasatake SugitaTakeshi KikuchiFumio HirataToru MiwaHiroki TakedaYorihisa OritaTakahiro SekiTomoko OhtaYuki KurauchiHiroshi KatsukiMuneaki MatsuoKatsumi HigakiKousaku OhnoShirou MatsumotoTakumi EraTetsumi IriePublished in: British journal of pharmacology (2021)
HP-γ-CD can serve as a fine-tuned cholesterol solubilizer for the treatment of NPC with a wider safety margin than HP-β-CD in terms of ototoxicity and pulmonary toxicity.