Induced pluripotent stem cell technology for disease modeling and drug screening with emphasis on lysosomal storage diseases
Journal
Stem Cell Research and Therapy
Journal Volume
3
Journal Issue
4
Date Issued
2012
Author(s)
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. ? 2012 BioMed Central Ltd.
SDGs
Other Subjects
agalsidase alfa; galsulfase; iduronate 2 sulfatase; laronidase; mercaptamine; miglustat; recombinant glucan 1,4 alpha glucosidase; sphingomyelin phosphodiesterase; carcinogenesis; cell differentiation; Chediak Higashi syndrome; cystinosis; drug efficacy; drug safety; drug screening; enzyme replacement; Fabry disease; fucosidosis; Gaucher disease; globoid cell leukodystrophy; glycogen storage disease type 2; graft rejection; high throughput screening; human; Hunter syndrome; Hurler syndrome; immunogenicity; lysosome storage disease; metachromatic leukodystrophy; mucolipidosis type 2; mucopolysaccharidosis type 7; neuronal ceroid lipofuscinosis; Niemann Pick disease; nonhuman; pluripotent stem cell; priority journal; protein homeostasis; review; Sanfilippo syndrome; somatic cell; teratoma; animal; biological therapy; cytology; disease model; fibroblast; Lysosomal Storage Diseases; pluripotent stem cell; transplantation; Animals; Cell- and Tissue-Based Therapy; Disease Models, Animal; Enzyme Replacement Therapy; Fibroblasts; Humans; Induced Pluripotent Stem Cells; Lysosomal Storage Diseases
Type
review