FIELD: biotechnology.
SUBSTANCE: described is a group of inventions including a modulating polynucleotide for reducing or inhibiting the expression of the SOD1 gene in a cell (variants), a viral genome of an adeno-associated virus (AAV) for reducing or inhibiting the expression of the SOD1 gene in a cell, wherein the viral AAV genome contains a nucleic acid sequence encoding the above modulating polynucleotide, a recombinant adeno-associated virus (rAAV) for reducing or inhibiting the expression of the SOD1 gene in a cell, a pharmaceutical composition containing rAAV for reducing or inhibiting the expression of the SOD1 gene in a cell, a method of reducing or inhibiting the expression of the SOD1 gene in a cell of a mammal, a method of treating amyotrophic lateral sclerosis (ALS) in a subject by reducing or inhibiting the expression of the SOD1 gene in a cell.
EFFECT: invention expands the range of means of inhibiting the expression of the SOD1 gene in a cell.
26 cl, 13 dwg, 19 tbl, 19 ex
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