Gene-modified neural progenitor cells for the treatment of neuropathic lysosomal storage diseases
Oriana Mandolfo
Brian W.Bigger
Stem Cell and Neurotherapies,Faculty of Biology,Medicine and Health,University of Manchester,Manchester,UK
摘要：Lysosomal storage diseases:Lysosomal storage diseases(LSDs)are a family of about 70 disorders,with an overall incidence of 1:7000 live births.They are caused by dysfunctional lysosomal hydrolases,eventually leading to the accumulation of undegraded substrate into the lysosome.This 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 decline.The majority of these diseases have a neurological component and in the absence of treatment,death often occurs in the first decades of life,with the neurological complications drastically undermining the patient's quality of life,as well as their families(Boustany,2013).
机标关键词：storagecellsdiseasesgene-modifiedlysosomalneuralneuropathicprogenitor
论文发表日期：2023-09-28
在线出版日期：2025-08-15（本平台首次上网日期，不代表文献的发表时间）
页数：2( 1954-1955 )