TITLE
Cell lines derived from Huntington’s disease induced Pluripotent Stem Cells
JHU REF. #
C14273
ABSTRACT
Cell models of Huntington's disease (HD) have been very important for understanding the cell biology of the disorder and for testing potential therapeutics The recent development of human induced pluripotent stem cell (iPSC) models has greatly enhanced the ability to model disease in human cells. Researchers at Johns Hopkins has generated immortalized striatal precursors from HD iPS cells as a new model for HD. These cells should be very valuable for studying HD pathogenesis and for screening for novel neuroprotective therapeutics
FEATURES
Johns Hopkins researchers have developed methods to immortalize the differentiating HD iPS cells at the stage of striatal precursors. The resulting cells are:
Easier to maintain and differentiate in medium spiny neurons in two weeks rather than three months required by previous model.
The HD neurons remain selectively venerable to neuronal degeneration.
Can be rescued by similar interventions as the parent iPSCs.
Assays can be performed in 96 well plates.
The cells can be screened for novel small molecules that can rescue toxicity and potentially could be developed into drugs to treat HD.
STAGE OF DEVELOPMENT
Tangible material, Disease-specific cell lines (iPS cell line model for HD).
DISEASE INDICATION
Huntington’s Disease
ASSOCIATED PUBLICATIONS
- Hum Mol Genet. 2014 Sep 15;23(R1):R17-26 -
