Article abstract


Nature Neuroscience 10, 608 - 614 (2007)
Published online: 15 April 2007 | doi:10.1038/nn1885

Non–cell autonomous effect of glia on motor neurons in an embryonic stem cell–based ALS model

Francesco Paolo Di Giorgio1,2,3,4, Monica A Carrasco2,4, Michelle C Siao2, Tom Maniatis2 & Kevin Eggan1,2


Here we report an in vitro model system for studying the molecular and cellular mechanisms that underlie the neurodegenerative disease amyotrophic lateral sclerosis (ALS). Embryonic stem cells (ESCs) derived from mice carrying normal or mutant transgenic alleles of the human SOD1 gene were used to generate motor neurons by in vitro differentiation. These motor neurons could be maintained in long-term coculture either with additional cells that arose during differentiation or with primary glial cells. Motor neurons carrying either the nonpathological human SOD1 transgene or the mutant SOD1G93A allele showed neurodegenerative properties when cocultured with SOD1G93A glial cells. Thus, our studies demonstrate that glial cells carrying a human SOD1G93A mutation have a direct, non–cell autonomous effect on motor neuron survival. More generally, our results show that ESC-based models of disease provide a powerful tool for studying the mechanisms of neural degeneration. These phenotypes displayed in culture could provide cell-based assays for the identification of new ALS drugs.

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  1. The Stowers Medical Institute, the Harvard Stem Cell Institute. Harvard University, 7 Divinity Ave., Cambridge, Massachusetts 02138, USA.
  2. the Department of Molecular and Cellular Biology, Harvard University, 7 Divinity Ave., Cambridge, Massachusetts 02138, USA.
  3. Perugia Universitâ Piazza dell' Universitâ 1, Perugia 06100, Italy.
  4. These authors contributed equally to this work.

Correspondence to: Tom Maniatis2 e-mail: maniatis@mcb.harvard.edu

Correspondence to: Kevin Eggan1,2 e-mail: eggan@mcb.harvard.edu


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