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Discoveries may advance stem cell therapy for Parkinson's, cancer patients

Two studies in the Jan. 27, 2006 Cell have yielded evidence that could prove a boon for stem cell therapies aimed at patients with Parkinson's disease and those with compromised immune systems due to intensive cancer therapy or autoimmune disease, according to researchers. The basic findings in mice revealed critical factors that determine the fate of one type of nerve cell progenitor and that set bone marrow stem cells into action.

Researchers at the Karolinska Institutet in Sweden discovered a "master determinant" that turns embryonic stem cells into bona fide dopamine neurons, brain cells that degenerate in those with Parkinson's disease. The findings hold promise for the future of cell replacement therapy for the debilitating and incurable disease characterized by tremors, said study authors Thomas Perlmann and Johan Ericson. The results also underscore the general importance of a thorough understanding of development for producing authentic cells of a desired type from stem cells.

"The use of cell replacement therapy in the treatment of Parkinson's disease is fraught with many problems," Perlmann said. "However, clinical trials have provided important proof of principle that transplantation of dopamine neurons might work in patients."

"Ethical and practical issues associated with transplantation of fetal dopamine neurons to patients with Parkinson's disease has triggered intense interest in the possibility of the use of in vitro-engineered stem cells as an unlimited cellular source for transplantation," the researchers added.

To investigate whether the identification of determinants underlying the specification of dopamine neurons during development could be exploited for generating dopamine neurons from stem cells, the researchers first looked for genes expressed in the dopamine progenitor cells in the developing midbrains of embryonic mice.

The researchers uncovered two transcription factor proteins, Lmx1a and Msx1, tha
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Contact: Heidi Hardman
hhardman@cell.com
617-397-2879
Cell Press
26-Jan-2006


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