FIELD: biotechnology.
SUBSTANCE: two-chain ribonucleic acid (tcRNA) for AROS3 gene expression inhibition os described, where this tcRNA contains (a) a sense chain consisting of a nucleotide sequence GcuuAAAAGGGAcAGuAuudTsdT, and an antisense chain, consisting of a nucleotide sequence AAuACUGUCCCUUUuAAGCdTsdT; (b) a sense chain consisting of a nucleotide sequence GCUUAAAAGGGACAGUAUUdTsdT, and an antisense chain, consisting of a nucleotide sequence AAUACUGUCCCUUUUAAGCdTsdT, (c) a sense chain consisting of a nucleotide sequence GfcUfuAfaAfaGfgGfaCfaGfuAfuUfdTsdT, and an antisense chain, consisting of a nucleotide sequence aAfuAfcUfgUfcCfcUfuUfuAfaGfcdTsdT, or (d) a sense chain consisting of a nucleotide sequence of GcuuAAAAGGGAcAGuAuudTsdT, and an antisense chain, consisting of a nucleotide sequence AAuACuGUCCCuuuuAagcdTsdT, where nucleotidesdenoted by lowercase letters, are 2'-O-methyl nucleotides; Gf, Af, Uf and Cf are 2'-fluoro nucleotides; and s is a phosphorothioate bond. A pharmaceutical composition for APOC3 gene expression inhibition containing the said tcRNA is also described. Methods for APOC3 expression inhibiting in a cell and treatment of a disorder mediated by APOC3 expression, including application of the said tcRNA, are provided.
EFFECT: invention expands the arsenal of drugs designed to treat a disorder mediated by APOC3 expression.
12 cl, 4 dwg, 12 tbl, 7 ex
