NYC Healthcare News



Blocking abnormal movement of mutated human FUS gene also blocks ALS process

March 16, 2016

According to the National Institutes of Health, Amyotrophic Lateral Sclerosis, sometimes called Lou Gehrig's disease, is a rapidly progressive, invariably fatal neurological disease that attacks the nerve cells (neurons) responsible for controlling voluntary muscles. The disease belongs to a group of disorders known as motor neuron diseases, which are characterized by the gradual degeneration and death of motor neurons. Motor neurons are nerve cells located in the brain, brainstem, and spinal cord that serve as controlling units and vital communication links between the nervous system and the voluntary muscles of the body. Messages from motor neurons in the brain (called upper motor neurons) are transmitted to motor neurons in the spinal cord (called lower motor neurons) and from them to particular muscles. In ALS, both the upper motor neurons and the lower motor neurons degenerate or die, ceasing to send messages to muscles. Unable to function, the muscles gradually weaken, waste away (atrophy), and twitch (fasciculations). Eventually, the ability of the brain to start and control voluntary movement is lost.

"Our next goal is to identify other factors such as proteins or RNA that mutant forms of FUS target so that we can get more insights into the disease mechanisms" said Nicholas Lanson Jr., an LSUHSC research associate and first author of the paper.

The Robert Packard Center for ALS at Johns Hopkins generously funded Dr. Pandey's lab in developing a fruit fly model of ALS.

Source: Louisiana State University Health Sciences Center