Amyotrophic Lateral Sclerosis (ALS) is a neurogenerative disease that impacts the nerve cells of the brain and spinal cord causing muscles to weaken and affecting physical function. Researchers at Nationwide Children’s Hospital have created a therapy approach to reduce the expression of the superoxide dismutase 1 gene (SOD1). The gene therapy combines the usage of AAV small hairpin RNA sequences (shRNA) and U7 small nuclear RNA (snRNA). The C9ORF72 repeat expansion is the known leading cause of ALS. The AAV shRNA will downregulate the SOD1 gene and fight against the C9ORF72 expansion while the U7 snRNA will induce exon skipping of the SOD1 mRNA and C9ORF72 repeat expansion to create an out of frame skipping that will degrade the transcripts. Combining the two approaches into a single vector could create an efficient gene therapy for ALS while treating a larger patient subpopulation.