The Effects of Mutated SOD1 on Amyotrophic Lateral Sclerosis and how siRNA Treatment Could Lead to a Cure

Senior Capstone Experience by Nathaniel Neuland ’21

Submitted to the Department of Chemistry

Advised by Dr. James Lipchock

Description: Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s Disease, is an incurable muscular dystrophy disease. The degradation of motor neurons and central nervous system leads to declining physical ability until the lungs are no longer functional. Superoxide dismutase (SOD1) is an essential metalloenzyme found within motor neurons that degrades free oxygen radicals. The SOD1 A4V mutation is strongly linked to familial ALS cases. The aggregation and loss of structural integrity associated with this mutation allows for the major consequences associated with ALS. While there is currently no cure for ALS, siRNA treatment technology has shown promise in heterozygous individuals as a way to silence the SOD1 gene with the A4V mutation. The research on siRNA demonstrates that it has been successful in vitro on multiple mutations of SOD1. It can bring cell viability to nearly 100% of wildtype levels due to its gene suppressing capacity. The research examined presents a strong case for the possibility of future clinical trials of siRNA that have a considerable possibility to lead to a cure for Lou Gehrig’s Disease.

Read Nathaniel’s SCE below:

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