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Approximately 30,000 Americans have amyotrophic lateral sclerosis (ALS), a neurodegenerative condition often referred to as Lou Gehrig’s disease. Approximately, 5 to 10 percent of all ALS cases are inherited, the rest are sporadic.

Michael Gitcho, PhD, postdoctoral research associate, and Nigel Cairns, PhD, research associate professor of neurology and pathology and immunology, Washington University School of Medicine, St. Louis, have linked a mutation in a gene known as TDP-43 to the inherited form of ALS. If the researchers are able to mutate this gene in laboratory animals it will be the first time they will be able to model the sporadic form of ALS, which will open the door to better understanding of ALS and provide new treatments.

What researchers learn may also shed light on other neurodegenerative disorders. It has been noted that abnormal TDP-43 has also been found in patients with frontotemporal dementia, the second most common cause of early onset of dementia–after Alzheimer’s disease. It is possible, researchers suggest, that this protein may allow them to find a link among several important diseases and help develop new targets for therapeutic intervention.

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