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Human Stem Cells Provide A New Model For Lou Gehrig's Disease

Date:
February 24, 2009
Source:
The Company of Biologists
Summary:
Human stem cells are used to create motor neurons that carry familial ALS mutations. These human stem cell-derived neurons look and act like normal motor neurons, and, when carrying ALS mutations, the cells undergo changes similar to those seen in ALS. This study presents a new human-derived cell system to use in studying ALS and ALS therapies.

Motor neurons derived from embryonic stem cells mimic the progress of familial ALS. Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a devastating condition in which motor neuron degeneration causes progressive loss of movement and muscle tone, leading to death.

Overcoming the limited success of previous models, a report published in Disease Models & Mechanisms (DMM) describes how neurons can be derived from human stem cells, and engineered to mimic inherited ALS.

Researchers at the University of California Los Angeles developed an optimized protocol to generate motor neurons from human embryonic stem cells (ES cells), which express normal or mutant forms of the SOD-1 gene, which is linked to inherited, familial ALS. Resulting cells exhibit hallmark characteristics of motor nerve cells, and neurons expressing mutant SOD-1 display abnormalities typical of ALS. Defects included shortened cell projections and a reduced life span compared to cells containing the normal SOD-1 gene.

This human cell-derived model of ALS provides a new method of studying this disease and testing novel therapeutics. This is especially helpful as only one drug is approved to help slow ALS progression, and animal models currently used in drug development have had limited success. Additionally, this research may aid other gene-linked neurodegenerative diseases, as they too may benefit from studies in a human cell-derived model.

The report was written by Saravanan Karumbayaram, Theresa K. Kelly, Andres A. Paucar, Anne J.T. Roe, Joy A. Umbach, Andrew Charles, Harley I. Kornblum, and Martina Wiedau-Pazos at the David Geffen School of Medicine at the University of California Los Angeles, and Steven A. Goldman at the University of Rochester Medical Center in Rochester, NY. The report is published in the March/April issue of Disease Models & Mechanisms (DMM), a research journal published by The Company of Biologists, a non-profit based in Cambridge, UK.


Story Source:

The above story is based on materials provided by The Company of Biologists. Note: Materials may be edited for content and length.


Cite This Page:

The Company of Biologists. "Human Stem Cells Provide A New Model For Lou Gehrig's Disease." ScienceDaily. ScienceDaily, 24 February 2009. <www.sciencedaily.com/releases/2009/02/090223083144.htm>.
The Company of Biologists. (2009, February 24). Human Stem Cells Provide A New Model For Lou Gehrig's Disease. ScienceDaily. Retrieved July 23, 2014 from www.sciencedaily.com/releases/2009/02/090223083144.htm
The Company of Biologists. "Human Stem Cells Provide A New Model For Lou Gehrig's Disease." ScienceDaily. www.sciencedaily.com/releases/2009/02/090223083144.htm (accessed July 23, 2014).

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