Gene-modified neural progenitor cells for the treatment of neuropathic lysosomal storage diseases

作     者：Oriana Mandolfo Brian W.Bigger Oriana Mandolfo;Brian W.Bigger

作者机构：Stem Cell and NeurotherapiesFaculty of BiologyMedicine and HealthUniversity of ManchesterManchesterUK 

出 版 物：《Neural Regeneration Research》 (中国神经再生研究（英文版）)

年 卷 期：2023年第18卷第9期

页      面：1954-1955页

核心收录：

学科分类：0710[理学-生物学] 1002[医学-临床医学] 1001[医学-基础医学(可授医学、理学学位)] 100204[医学-神经病学] 10[医学] 

基　　金：funded by The Orphan Disease Centre,University of Pennsylvania support from Innovate UK (Innovate Manchester Advanced Therapy Centre Hub-iMATCH) 

主　　题：diseases progenitor eventually 

摘      要：Lysosomal storage diseases:Lysosomal storage diseases(LSDs) are a family of about 70 disorders,with an overall incidence of 1:7000 live *** are caused by dysfunctional lysosomal hydrolases,eventually leading to the accumulation of undegraded substrate into the *** results in a wide array of symptoms,which may include:the presence of dysmorphic features,cardio-respiratory disease,bone and joint disease,organomegaly,developmental delay and neurocognitive *** majority of these diseases have a neurological component and in the absence of treatment.