MIRNA STRUCTURES WITH HIGH ACTIVITY AND REDUCED EXPOSURE OUTSIDE THE TARGET Russian patent published in 2023 - IPC A61K31/713 A61K48/00 A61P35/00 C07H21/00 C12N15/113 

FIELD: medicine.

SUBSTANCE: proposed group of inventions relates to medicine, namely to pharmaceuticals. An RNAi molecule has been proposed for downregulating target mRNA expression, consisting of a polynucleotide sense strand and a polynucleotide antisense strand. The molecule has a duplex region 15 to 30 nucleotides in length, in which one or more nucleotides in the antisense strand at positions 3 to 8 from the 5'-end of the duplex region are deoxynucleotides, where the 5'-end of the antisense strand corresponds to the 5'-terminus a duplex region where the antisense strand has deoxynucleotides at a plurality of positions in the primer region of said antisense strand. Proposed pharmaceutical composition for the treatment of malignant tumors, containing the specified molecule and a pharmaceutically acceptable carrier. Proposed cell for the expression of the RNAi molecule containing the specified molecule. A method for treating malignant tumors in a subject by means of gene silencing is proposed. Proposed is the use of a pharmaceutical composition for the treatment of malignant tumors in a subject and the use of the composition for the preparation or manufacture of a drug for the treatment of malignant tumors in a subject in need thereof.

EFFECT: proposed group of inventions provides an increase in gene knockdown activity through the use of an RNAi molecule in which one or more deoxynucleotides are located in the siRNA primer region.

27 cl, 15 dwg, 22 tbl, 22 ex