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Stem Cells Hold Promise for Treating Neurodegenerative Diseases

A research finds that Human embryonic stem cells (hESCs) hold great promise for benefiting degenerative diseases. The study is the first to provide evidence// that stem cell-derived nerve cells may integrate electrically and functionally into a diseased brain.

This study was conducted 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.

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 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 gene that makes the housekeeping enzyme hexosaminidase ("hex") deficient and, therefore, has Sandhoff's Disease, a lethal genetic disease related to Tay-Sachs Disease. When stem cells were implanted -- at simply one time point -- into brains of newborn Sandhoff mice, the onset of symptoms was delayed, well-being and motor function was preserved, and lifes
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