FIELD: biotechnology.SUBSTANCE: invention refers to biotechnology and molecular biology. Disclosed is an adenoassociated virus (AAV)-based vector genome for inhibiting or suppressing superoxide dismutase 1 (SOD1) gene expression in a mammalian cell, as well as a corresponding particle, a method of inhibiting gene expression, a pharmaceutical composition, a method of treating amyotrophic lateral sclerosis and duplex siRNA.EFFECT: inventions can be used in medicine to expand the range of agents for controlling amyotrophic lateral sclerosis.23 cl, 12 dwg, 22 tbl, 18 ex
COMPOSITIONS AND METHODS OF TREATING AMYOTROPHIC LATERAL SCLEROSIS (ALS)