Surrounding Cells Prevent Damage from ALS Mutant Gene

Researchers working with a mouse model for amyotrophic lateral sclerosis (ALS) have found that surrounding neurons carrying the ALS mutant SOD1 gene with normal non-neuronal cells slows or prevents the onset of ALS symptoms.

Investigators at the University of California, San Diego (USA), genetically engineered a line of chimeric mice that carried the mutated gene of ALS in motor neurons but the normal gene in the surrounding non-neuronal tissue.

The study, which was published in the October 3, 2003, issue of Science, showed that even when as many as three-fourths of the motor neurons in the animals' spinal cords carried the mutant gene, all the motor neurons remained healthy, and survival of the animals was increased by one to eight months or more. On the other hand, mice with normal motor neurons but with surrounding cells carrying an SOD1 mutation showed early signs of disease.

"What we have been given is a new principle for extending survival or, perhaps, overcoming ALS, based on how many healthy cells surround an ailing motor nerve cell,” explained senior author Dr. Don Cleveland, a researcher at the University of California, San Diego. "All this has great implications for stem cell therapy. We now believe delivery of normal, non-neuronal cells to spinal cords could be completely protective, even without replacement of a single motor neuron.”



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