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
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