摘要:
The present disclosure relates to an RNAi-inducing nucleic acid molecule and use thereof. An aspect of the disclosure relates to an RNAi-inducing nucleic acid molecule for inhibiting expression of myeloid differentiation primary response gene 88 (MyD88). Another aspect of the present disclosure relates to a pharmaceutical composition for treating or preventing age-related macular degeneration, comprising the RNAi-inducing nucleic acid molecule.
摘要:
The present invention relates to a novel, RNAi-inducing nucleic acid molecule having cell penetrating ability and the use thereof, and more particularly, to a novel, RNAi-inducing double-stranded nucleic acid molecule, which has a replacement of the phosphate backbone of at least one nucleotide with phosphorothioate or phosphorodithioate, and has a lipophilic compound conjugated thereto, and thus has high target gene-silencing efficiency while having the ability to penetrate cells without needing a separate intracellular delivery vehicle, and to a method of silencing a target gene using the nucleic acid molecule. The nucleic acid structure according to the present invention has both cholesterol modification and phosphorothioate modification introduced therein, and thus has high gene silencing efficiency while having the ability to penetrate cells without needing a separate intracellular delivery vehicle. Thus, it can be delivered into an actual target area in an amount sufficient for induction of RNAi, and thus can overcome the in vivo delivery problem occurring in the prior art. Therefore, the nucleic acid molecule according to the invention can effectively substitute for conventional siRNA molecules to treat cancer or viral infections.
摘要:
The present application relates to an RNA interference (RNAi) agent for inhibiting the expression of hepatitis B virus and uses thereof, and to an RNAi agent that inhibits the expression of hepatitis B virus and a pharmaceutical composition including the RNAi agent for amelioration or treatment of diseases caused by hepatitis B virus infection.
摘要:
The present invention relates to an asymmetric siRNA that may inhibit expression of programmed cell death protein 1 (PD-1), and a use thereof, and more specifically, to: an asymmetric siRNA including an antisense strand including a sequence complementary to mRNA encoding PD-1, and a sense strand forming complementary bonds with the antisense strand; a pharmaceutical composition for preventing or treating cancer, including the asymmetric siRNA; an immune cell in which expression of PD-1 is inhibited, obtained by treatment with the siRNA; and an immune cell therapeutic agent for treating cancer, including the immune cell.
摘要:
In certain aspects, provided herein are RNA complexes that inhibit Myeloid differentiation primary response gene 88 (MyD88) and/or Toll-like receptor 3 (TLR3) and are useful in the treatment of age-related macular degeneration (AMD). In certain aspects, provided herein are pharmaceutical compositions comprising such RNA complexes and methods of using such RNA complexes and pharmaceutical compositions.
摘要:
The present invention relates to a novel, RNAi-inducing nucleic acid molecule having cell penetrating ability and the use thereof, and more particularly, to a novel, RNAi-inducing double-stranded nucleic acid molecule, which has a replacement of the phosphate backbone of at least one nucleotide with phosphorothioate or phosphorodithioate, and has a lipophilic compound conjugated thereto, and thus has high target gene-silencing efficiency while having the ability to penetrate cells without needing a separate intracellular delivery vehicle, and to a method of silencing a target gene using the nucleic acid molecule. The nucleic acid structure according to the present invention has both cholesterol modification and phosphorothioate modification introduced therein, and thus has high gene silencing efficiency while having the ability to penetrate cells without needing a separate intracellular delivery vehicle. Thus, it can be delivered into an actual target area in an amount sufficient for induction of RNAi, and thus can overcome the in vivo delivery problem occurring in the prior art. Therefore, the nucleic acid molecule according to the invention can effectively substitute for conventional siRNA molecules to treat cancer or viral infections.
摘要:
The present application relates to an asymmetric RNAi-inducing nucleic acid molecule which inhibits the expression of retinoid-related orphan nuclear receptor-beta (ROR-beta) and use thereof, and more particularly to an asymmetric RNAi-inducing nucleic acid molecule comprising an antisense strand comprising a sequence complementary to mRNA encoding ROR-beta, and a sense strand forming complementary bonds with the antisense strand, and a pharmaceutical composition for ameliorating or treating a retinal disease, comprising the asymmetric RNAi-inducing nucleic acid molecule.
摘要:
The present invention relates to an RNAi-inducing nucleic acid molecule having a new structure and the use thereof, and more particularly to a novel nucleic acid molecule having a structure comprising a first strand, which is 24-121 nt in length and comprises a region complementary to a target nucleic acid, and a second strand which is 13-21 nt in length and has a region that binds complementarily to the region of the first strand, which is complementary to the target nucleic acid, so that the nucleic acid molecule inhibits the expression of a target gene with increased efficiency, and to a method of inhibiting the expression of a target gene using the nucleic acid molecule. The nucleic acid molecule structure of the present invention increases the efficiency with which the nucleic acid molecule inhibits the target gene. Alternatively, the nucleic acid molecule of the present invention can either increase the ability of the siRNA to bind to the target gene or cause synergistic cleavage, by introduction of antisense DNA, antisense RNA, ribozyme or DNAzyme, thereby increasing the efficiency with which the nucleic acid molecule inhibits the target gene. In addition, when the nucleic acid molecule according to the present invention is used, the efficiency with which the target gene is inhibited can be maintained for an extended period of time. Accordingly, the RNAi-inducing nucleic acid molecule of the present invention can be effectively used for the treatment of cancer or viral infection in place of conventional siRNA molecules.
摘要:
The present invention relates to an asymmetric siRNA which inhibits an expression of male pattern hair loss target genes and a use thereof and, more particularly, to an asymmetric siRNA which inhibits an expression of 3-oxo-5-alpha-steroid-4-dehydrogenase 1 (SRD5A1) gene, 3-oxo-5-alpha-steroid-4-dehydrogenase 2 (SRD5A2) gene or androgen receptor (AR) gene, and a composition for prevention or treatment of hair loss comprising the asymmetric siRNA.