, Volume 8, Issue 4, pp 659-667

First online:

Neural Stem Cell Transplantation as a Therapeutic Approach for Treating Lysosomal Storage Diseases

  • Lamya S. ShihabuddinAffiliated withGenzyme Corporation Email author 
  • , Seng H. ChengAffiliated withGenzyme Corporation


Treating the central nervous system manifestations of subjects with neuropathic lysosomal storage diseases remains a major technical challenge. This is because of the low efficiency by which lysosomal enzymes in systemic circulation are able to traverse the blood brain barrier into the central nervous system. Intracranial transplantation of neural stems cells genetically modified to overexpress the respective deficient enzymes represents a potential approach to addressing this group of diseases. The unique properties of neural stem cells and progenitor cells, such as their ability to migrate to distal sites, differentiate into various cell types and integrate within the host brain without disrupting normal function, making them particularly attractive therapeutic agents. In addition, neural stem cells are amenable to ex vivo propagation and modification by gene transfer vectors. In this regard, transplanted cells can serve not only as a source of lysosomal enzymes but also as a means to potentially repair the injured brain by replenishing the organ with healthy cells and effecting the release of neuroprotective factors. This review discusses some of the well-characterized neural stem cell types and their possible use in treating neuropathic lysosomal storage diseases such as the Niemann Pick A disease.


Neural stem cells Neural progenitor cells Cell transplantation Lysosomal storage diseases Niemann Pick A disease