Stem cell-based therapy for lysosomal storage diseases

Brittni A. Scruggs, Xiujuan Zhang, Jeffrey M. Gimble, Bruce A. Bunnell

Research output: Chapter in Book/Report/Conference proceedingChapterpeer-review

2 Scopus citations


The family of lysosomal storage diseases (LSDs) affects 1 out of every 5,000-7,000 live births, and most affected LSD patients experience a rapidly progressive course after disease onset due to dysfunction of the lysosome. The brain is particularly sensitive to the abnormal storage of lysosomal aggregates, and lysosomal dysfunction in neurons and other cell types leads to the progressive degeneration of the central nervous system (CNS) in as many as 75% of all LSDs. Transplantation of stem cells has been studied for several LSDs due to the tremendous promise of these cells to deliver therapeutic gene products, sequester toxic compounds (e.g., accumulated metabolites), replace affected cells through engraftment and differentiation, and decrease inflammation in neurodegenerative disease states. This chapter describes the LSDs that have been tested using stem cell transplantation (SCT), and a review of the current state of LSD research is presented and highlights clinical improvements that have been reported.

Original languageEnglish
Title of host publicationStem Cells and Human Diseases
PublisherSpringer Netherlands
Number of pages30
ISBN (Electronic)9789400728011
ISBN (Print)940072800X, 9789400728004
StatePublished - 1 Apr 2012


  • Embryonic stem cells
  • Hematopoietic stem cell transplantation
  • Lysosomal storage disease
  • Mesenchymal stem cells
  • Neural stem cells
  • Stem cell-based therapy


Dive into the research topics of 'Stem cell-based therapy for lysosomal storage diseases'. Together they form a unique fingerprint.

Cite this