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

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).