Washington, Feb 27 (ANI): Scientists have described a new human cell-derived model of myotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, which can offer new method of studying the disease and testing novel therapeutics.
Lou Gehrig's disease is a devastating condition in which motor neuron degeneration causes progressive loss of movement and muscle tone, leading to death.
And in order to overcome the limited success of previous models, researchers at the University of California Los Angeles have now described how neurons can be derived from human stem cells, and engineered to mimic inherited ALS.
The researchers developed an optimised 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.
The cells, formed a result, 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.
The new model 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.
Also, the research may aid other gene-linked neurodegenerative diseases, as they too may benefit from studies in a human cell-derived model.
The study has been published in Disease Models and Mechanisms (DMM), dmm.biologists.org. (ANI)