关于脑内灌注Cerliponase alfa治疗CLN2疾病的综述  

Intracerebral Perfusion of Cerliponase Alfa in the Treatment of CLN2 Disease

作　　者：王丹[1] 孙婷婷 孟雪莲[1] 陈长兰[1] WANG Dan;SUN Ting-ting;MENG Xue-lian;CHEN Chang-lan(College of pharmacy,LiaoningUniversity,Shenyang 110036,China)

机构地区：[1]辽宁大学药学院

出　　处：《辽宁大学学报（自然科学版）》2019年第3期237-243,共7页Journal of Liaoning University：Natural Sciences Edition

基　　金：辽宁省自然科学基金指导计划(20180551180)

摘　　要：迟发性婴儿型2型神经元蜡样脂褐质沉积症(late infantile neuronal ceroid lipofuscinosis type 2,CLN2)又称为三肽基肽酶-1(TPP1)缺乏症,是Batten病的一种.Cerliponase alfa(Brineura^TM)是一种用于治疗CLN2的药物.文章综述了CLN2的病理特征、TPP1酶和cerliponase alfa(Brineura^TM)的使用方法和注意事项,为CLN2疾病的进一步研究提供参考.Late infantile neuronal ceroid lipofuscinosis type 2(CLN2)is also known as three peptidyl peptidase-1(TPP1)deficiency,one of the Batten diseases.Cerliponase alfa(Brineura^TM)is a drug used to treat CLN2.This article reviews the pathological features of CLN2,the use of TPP1 enzyme and cerliponase alfa(Brineura^TM),and points for attention,which will provide reference for further research on CLN2 disease.

关 键 词：cerliponase alfa(Brineura TM) CLN2 TPP1酶 酶替代疗法 脑室灌注 

分 类 号：R965[医药卫生—药理学]