RNA MOLECULES Russian patent published in 2024 - IPC C12N15/113 C12N15/86 

FIELD: biotechnology.

SUBSTANCE: invention is an RNA molecule for downregulating the level and/or activity of a target RNA molecule, where the RNA molecule includes a first RNA component, a second RNA component which is covalently bonded to the first RNA component, and, optionally, one or more, or all of: (i) a connecting ribonucleotide sequence which covalently binds the first and second RNA components, (ii) 5'-leader sequence and (iii) 3'-trailer sequence, where the first RNA component consists, in order from 5' to 3', of first 5'-ribonucleotide, a first RNA sequence and first 3'-ribonucleotide, where first 5' and 3'-ribonucleotides are paired with each other in the first RNA component, where the first RNA sequence includes a first sense ribonucleotide sequence of at least 20 consecutive ribonucleotides, a first loop sequence of at least 4 ribonucleotides and a first antisense ribonucleotide sequence of at least 20 consecutive ribonucleotides, where the first antisense ribonucleotide sequence is hybridised with the first sense ribonucleotide sequence in the RNA molecule, where the first antisense ribonucleotide sequence is capable of hybridisation with the first region of the target RNA molecule, where the second RNA component is covalently bonded through the connecting ribonucleotide sequence, if present, or directly, if the connecting ribonucleotide sequence is absent, with first 5'-ribonucleotide or first 3'-ribonucleotide, where the second RNA component consists, in order from 5' to 3', from second 5'-ribonucleotide, a second RNA sequence and second 3'-ribonucleotide, where second 5' and 3'-ribonucleotides are paired with each other in the RNA molecule, where the second RNA sequence includes a second sense ribonucleotide sequence, a second loop sequence of at least 4 ribonucleotides and a second antisense ribonucleotide sequence, wherein the second sense ribonucleotide sequence is hybridised with a second antisense ribonucleotide sequence in the RNA molecule, where 5'-leader sequence, if present, consists of a sequence of ribonucleotides covalently bonded to first 5'-ribonucleotide, if the second RNA component is linked to first 3'-ribonucleotide, or to second 5'-ribonucleotide, if the second RNA component is linked to first 5'-ribonucleotide, and where 3'-trailer sequence, if present, consists of a sequence of ribonucleotides covalently bonded to second 3'-ribonucleotide, if the second RNA component is linked to first 3'-ribonucleotide, or to first 3'-ribonucleotide, if the second RNA component is linked to first 5'-ribonucleotide.

EFFECT: invention makes it possible to effectively implement a method for downregulation of the level and/or activity of a target RNA molecule in the body by delivering an RNA molecule into the body according to the invention.

92 cl, 37 dwg, 1 tbl, 20 ex