Scientists with Cedars-Sinai Medical Center are building an improved stem-cell model of amyotrophic lateral sclerosis (ALS) – also known as Lou Gehrig’s disease – to accelerate progress toward developing a cure for the devastating neurological disorder. Their findings demonstrate that current models can be enhanced to show how ALS damages cells later in life.
ALS affects more than 12,000 people in the U.S., according to the National Institutes of Health. The disease typically affects people ages 40 and 70, according to the ALS Association. There are no effective treatments, and most patients suffer paralysis and die within five years of diagnosis.
Researchers at the Cedars-Sinai Board of Governors Regenerative Medicine Institute are creating induced pluripotent stem cells (iPSCs) derived from patients’ skin cells. By altering genes in the cells, they can be returned to their embryonic, or stem cell state. The specially engineered stem cells can be reproduced indefinitely in a laboratory, where their abnormalities can be examined in detail.
A multi-center team led by Clive Svendsen, Ph.D. and Ritchie Ho, Ph.D. has found that investigators need to devise ways to alter iPSC versions of spinal motor neurons to more closely resemble older versions found in adult ALS patients, the scientists said. Using motor neurons to model neurological disease may require aging them in a dish, said Ho, the first author of an article on the study, published online on July 18 in the journal “Nature Neuroscience.” To help researchers age the stem cells, the group identified a series of genetic markers to assess cell maturity and age of cells.
“By knowing the gene expression patterns that define adult motor neurons in the spinal cord, we can push the iPSC-derived motor neurons in the right direction in the petri dish,” said Svendsen, director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute and professor of Medicine and Biomedical Sciences. Svendsen was the senior author of the article.
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