Resource: Library of ALS Patients-Derived Stem Cells Now Open to Researchers

Although rodent models of ALS have been a valuable tool for unraveling disease mechanisms, positive results in these models have thus far not translated into effective therapeutics in humans. Recently developed methods for deriving stem cells from ALS patients are providing a promising new avenue to understand molecular pathways in ALS and to screen for drugs effective at modulating those pathways (see April 2015 news story). Now, researchers led by Jeffrey Rothstein from the Johns Hopkins University in Baltimore have created the largest public repository of induced pluripotent stem cells (iPSC) from familial ALS (fALS) patients, described in detail in the March 11 PLOS ONE (Yi, L., et. al., 2015). The library includes cell lines derived from 22 fALS patients carrying mutations in the most common fALS linked genes, including SOD1, C9ORF72, and FUS. These iPSCs can be differentiated into astroglia, which have been implicated as central players in non-cell autonomous mechanisms mediating ALS disease progression (See Feb 2014 news story). This valuable resource for research and drug screening is available through the Coriell Institute to researchers from both academia and commercial entities.

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disease-als human tissue and iPSCs topic-preclinical
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