by Michelle AuCoin and Hans S. Keirstead, Ph.D. (From FSMA newsletter, Winter 2005)

Although treatment can ease complications of SMA, no cure exists. Still, the NIH has deemed SMA one of the neurological genetic conditions closest to an effective treatment or cure in the near future. To date, the primary approaches to treating or curing SMA have focused on two strategies. First, genetic therapy – manipulating the genetic material responsible for producing SMA. Second, cellular replacement therapy – replacing dead or dying motor neurons with new ones.

Human embryonic stem cells (hESCs) offer great promise for cellular replacement strategies due to their ability to generate every cell in the body (every human is made from their hESCs), and their seemingly unlimited ability to replicate themselves (allowing for huge numbers of cells to be generated). The ability to amplify hESCs to enormous numbers already exists. Generating medically useful cell populations from hESCs has been one of the largest obstacles facing hESC researchers. How do we coax hESCs to become the one cell type that we desire for treatment of a human disease? For the first time, the Keirstead Research Group at University of California at Irvine has produced high purity cells from hESCs.
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