Induced pluripotent stem cell technology for disease modeling and drug screening with emphasis on lysosomal storage diseases

Stem Cell Res Ther. 2012 Aug 28;3(4):34. doi: 10.1186/scrt125.

Abstract

The recent derivation of disease-specific induced pluripotent stem cells (iPSCs) from somatic cells of patients with familial and sporadic forms of diseases and the demonstration of their ability to give rise to disease-relevant cell types provide an excellent opportunity to gain further insights into the mechanisms responsible for the pathophysiology of these diseases and develop novel therapeutic drugs. Here, we review the recent advances in iPSC technology for modeling of various lysosomal storage diseases (LSDs) and discuss possible strategies through which LSD-iPSCs can be exploited to identify novel drugs and improve future clinical treatment of LSDs.

Publication types

  • Research Support, U.S. Gov't, Non-P.H.S.
  • Review

MeSH terms

  • Animals
  • Cell- and Tissue-Based Therapy
  • Disease Models, Animal
  • Enzyme Replacement Therapy
  • Fibroblasts / cytology
  • Humans
  • Induced Pluripotent Stem Cells / cytology
  • Induced Pluripotent Stem Cells / transplantation*
  • Lysosomal Storage Diseases / therapy*