Penta-ALS Platform for Amyotrophic Lateral Sclerosis

Disease Background: Amyotrophic lateral sclerosis (ALS) also called as a motor neuron disease is characterized by the presence of inclusion bodies in the cytoplasm of motor neurons. A number of pathological proteins have been associated with the inclusion bodies namely TDP-43, MST1, SOD1, FUS, and C9orf72. The most common cause of death in ALS patients is respiratory failure.

 

Details on the Model: Pentagrit zebrafish model of amyotrophic lateral sclerosis is a TDP-43 mutant that replciates the death of pyramidal neurons in the motor cortex and motor neurons in the brain stem and central spinal environment. Eventually the animal develops muscle atrophy with symptoms initiating with stiffening muscles and muscle twitching, affecting activities such as swallowing, and breathing. Pathological hallmarks includes the sclerosis of the corticospinal and corticobulbar tracts with thinning of the hypoglossal nerves and anterior roots of the spinal cord. Riluzole the first treatment for amyotrophic lateral sclerosis to approved by the U.S. Food and Drug Administration employed as a comparator shows neuroprotective and rescue of motor activity of zebrafish model of ALS. Similarly edarvone as a comparator rescues oxidative stress driven degeneration of both motor neurons and skeletal muscles in zebrafish model of amyotrophic lateral sclerosis