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Stem cells act through multiple mechanisms to benefit mice with neurodegenerative disease

Human embryonic stem cells (hESCs) hold great promise for benefiting degenerative diseases, and do so by invoking multiple mechanisms. Such cells can be grown in a manner compatible with clinical use (i.e., without animal feeder layers) and even without the need for immunosuppression. These were a few of a number of conclusions arrived at by an international collaboration led by Evan Y. Snyder, M.D., Ph.D., and spearheaded by a member of his lab, Jean-Pyo Lee, Ph.D., of the Burnham Institute for Medical Research ("Burnham"). The study, to be published in Nature Medicine, will be made available by advanced publication at the journal's website on March 11, 2007.

To determine whether stem cell biology might play a role in benefiting degenerative diseases, the investigators first chose to approach, as proof-of-concept, a mouse model of a representative lethal neurodegenerative disease. Next, they used mouse neural stem cells (NSCs), a type of "adult" stem cell, to establish the parameters of what might or might not be achievable in this disease. Then, having demonstrated success with mouse cells, they extended those insights to stem cells of human origin, both human neural stem cells and human embryonic stem cells, and, in fact, had the opportunity, for the first time, to compare those two types of controversial stem cells head-to-head in the same model. The results, described in more detail below, in fact prove to be the first successful use of human embryonic stem cells in treating a degenerative disease, significantly preserving function and extending life.

The mouse model chosen falls in a class of genetic diseases that afflicts 1 in 5000 patients, typically children (called lysosomal storage diseases, described in more detail below), but which is often used to model an array of adult neurodegenerative diseases such as Parkinson's, ALS, Alzheimer's - particularly those with a genetic component. The mouse used here has mutation in a ge
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Source:Burnham Institute


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