The expanding world of stem cell modeling of Huntington's disease: creating tools with a promising future.

Journal: 
Genome Med
Publication Year: 
2012
Authors: 
Jacqueline M Ward , Albert R La Spada
Public Summary: 
Scientific Abstract: 
ABSTRACT: Deconstructing the mechanistic basis of neurodegenerative disorders, such as Huntington's disease (HD), has been a particularly challenging undertaking, relying mostly on post-mortem tissue samples, non-neural cell lines from affected individuals, and model organisms. Two articles recently published in Cell Stem Cell report first the generation and characterization of induced pluripotent stem cell (iPSC)-derived models for HD, and second, the genetic correction of a disease-causing CAG expansion mutation in iPSCs from individuals with HD. Taken together, these two studies provide a framework for the production and validation of iPSC materials for human neurodegenerative disease research and yield crucial tools for investigating future therapies.

© 2013 California Institute for Regenerative Medicine