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Aug 7 2008, 1:18 PM EST
Pluripotent Stem Cells Created for 10 Diseases
GEN News Highlights
A group of scientists have created 10 disease-specific stem cell strains. 
They will make the cell lines available worldwide through a facility funded 
by the Harvard Stem Cell Institute.
The team developed these stem cell lines for Duchenne muscular dystrophy, 
Becker muscular dystrophy, juvenile-onset (type I) diabetes, Parkinson's 
disease, Huntington's disease, Down's syndrome, ADA severe combined 
immunodeficiency, Shwachman-Bodian-Diamond syndrome, Gaucher disease, and 
Lesch-Nyhan syndrome.
In many cases these new stem cell cultures will mimic human disease more 
reliably than animal models, according to Howard Hughes Medical Institute 
investigator George Q. Daley, who led the work. The technique will even 
enable the comparison of how the same disease varies among people, by 
generating disease-specific stem cell cultures from many individuals. The 
cells will also offer a proving ground for screening drugs to treat disease.
To produce the disease-specific stem cells, the researchers mixed cells from 
patients with the disorders with benign viruses to introduce certain 
previously discovered reprogramming factors into the cells.
Once the investigators isolated the disease-specific stem cells, they 
analyzed the genes and confirmed that the stem cells had the same 
disease-causing defects as the original donor cells. The researchers also 
made sure that the stem cells were pluripotent.
Scientists from Harvard Medical School, Massachusetts General Hospital, and 
the University of Washington were also involved in this study. Their results 
appear in an advanced online August 7 publication of Cell.

Rayilyn Brown
Director AZNPF
Arizona Chapter National Parkinson Foundation
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