Abstract: An siRNA conjugate having a structure as represented by formula (1) for inhibiting hepatitis B vims gene expression, comprising siRNA and a conjugated group, wherein the sense strand of the siRNA comprises a nucleotide sequence 1, and the antisense strand comprises a nucleotide sequence 2; the nucleotide sequence 1 and the nucleotide sequence 2 are, at least in part, reversely complementary to form a double-stranded region; the nucleotide sequence 1 and SEQ ID NO: 1 are equal in length and differ by no more than three nucleotides; the nucleotide sequence 2 and SEQ ID NO: 2 are equal in length and differ by no more than three nucleotides. The siRNA conjugate can specifically target liver cells and effectively solve the problem of siRNA delivery in vivo, and shows excellent activity and low toxicity to inhibit HBV gene expression while maintaining high stability of siRNA.

Abstract: Provided is a siRNA for inhibiting HBV gene expression, including a sense strand and an antisense strand. The sense strand includes a nucleotide sequence 1, and the antisense strand includes a nucleotide sequence 2; the nucleotide sequence 1 and the nucleotide sequence 2 are at least partially reversely complementary to form a double-stranded region; the nucleotide sequence 1 and the nucleotide sequence shown in SEQ ID NO: 1 are equal in length, and no more than 3 nucleotide differences are generated; the nucleotide sequence 2 and the nucleotide sequence shown in SEQ ID NO: 2 are equal in length, and no more than 3 nucleotide differences are generated; nucleotides of the 7th, 8th, and 9th bits of the nucleotide sequence 1 and the 2nd, 6th, 14th, and 16th of the nucleotide sequence 2 are fluoro-modified nucleotides. Also provided are a pharmaceutical composition and a conjugate containing the siRNA.

Abstract: Provided are an siRNA for inhibiting expression of a Hepatitis B virus gene, and a pharmaceutical composition and conjugate containing the siRNA. Each nucleotide in the siRNA is an independently modified or unmodified nucleotide; the siRNA comprises a sense strand and an antisense strand; the sense strand comprises a nucleotide sequence A; the length of the nucleotide sequence A is the same as that of a nucleotide sequence as shown in SEQ ID NO: 1, and the number of the nucleotide differences is not more than three; the antisense strand comprises a nucleotide sequence B; and the length of the nucleotide sequence B is the same as that of a nucleotide sequence as shown in SEQ ID NO: 2, and number of nucleotide differences is not more than three.

Abstract: Provided are a siRNA for inhibiting the expression of an angiopoietin-like protein 3 (ANGPTL3) gene, and a pharmaceutical composition and a conjugate comprising the siRNA; wherein each nucleotide in the siRNA is independently a modified or unmodified nucleotide, and the siRNA comprises a sense strand and an antisense strand; the sense strand comprises a nucleotide sequence A, the nucleotide sequence A having the same length as the nucleotide sequence as represented by SEQ ID NO:1 with no more than 3 nucleotide differences; the antisense strand comprises a nucleotide sequence B, the nucleotide sequence B having the same length as the nucleotide sequence as represented by SEQ ID NO:2 with no more than 3 nucleotide differences.

Abstract: The present disclosure provides a siRNA for inhibiting the expression of apolipoprotein C3 (ApoC3) gene, and a pharmaceutical composition and a conjugate comprising the siRNA; wherein each nucleotide in the siRNA is independently a modified nucleotide, and the siRNA comprises a sense strand and an antisense strand; the sense strand comprises a nucleotide sequence A, the nucleotide sequence A having the same length as the nucleotide sequence as represented by SEQ ID NO:1 with no more than 3 nucleotide differences; the antisense strand comprises a nucleotide sequence B, the nucleotide sequence B having the same length as the nucleotide sequence as represented by SEQ ID NO:2 with no more than 3 nucleotide differences.

Abstract: Disclosed are a small interfering nucleic acid specifically inhibiting HBV gene expression, and a pharmaceutical composition and a siRNA conjugate which contain the small interfering nucleic acid and can be used for preventing and/or treating hepatitis B. Also provided is a use of the small interfering nucleic acid in preparing drugs for preventing and/or treating hepatitis B.

Abstract: The present invention provides siRNAs for inhibiting the expression of plk1 gene, and the method for inhibiting the expression of plk1 gene in mammalian cells. The siRNAs of the present invention have the double-stranded structure, and said double-stranded structure is composed of the first single strand and the second single strand that are fully complementary, wherein the sequence of said first single strand is the same as the target sequence within the sequence as shown in SEQ ID NO: 1, and the sequence of said second single strand is complementary to the target sequence within the sequence as shown in SEQ ID NO: 1. The siRNAs of the present invention can sequence specifically mediate the inhibition of plk1 gene expression, and have a good serum stability. By the introduction of the siRNAs of the present invention into the tumor cells, the expression of plk1 gene can be effectively inhibited, and the growth of tumor cells is inhibited and the apoptosis of tumor cells is promoted.

Abstract: Provided are a polycaprolactone-polyphosphate block copolymer, a liquid composite formed by the block copolymer, a nucleic acid preparation, preparation methods for the copolymer and the liquid composite, and the use of the copolymer and the liquid composite in a nucleic acid medicine delivery system. The block copolymer prepared using the present invention has good biocompatibility, low cytotoxicity, and good biodegradability. The micelles provided in the present invention self-assemble into nano-particles in an aqueous solution, and have good stability, biocompatibility, and biodegradability, and low cytotoxicity. The preparation method is simple, has high repeatability, as a vector can protect small nucleic acids such as siRNA from biodegradation, can combine with the scale effect of nano-particles, and can be used for treating different diseases. Additionally, bonding targeting groups enable specificity recognition of different cancer cells.

Abstract: Provided are a double-stranded nucleic acid and use thereof in ribonuclease detection, and a ribonuclease detection method and use thereof in tumor detection and/or diagnosis. Specifically, a double-stranded nucleic acid substrate comprises at least one ribonuclease sensitive site. The activity and content of the ribonuclease in a sample are detected by analyzing the degradation of the double-stranded nucleic acid substrate by the ribonuclease. Also provided are a ribonuclease detection kit and a tumor detection kit.

Abstract: Provided are a small interfering nucleic acid against bone formation inhibiting gene CKIP-1, a pharmaceutical composition thereof, and uses thereof in preparation of a pharmaceutical composition for treating and/or preventing diseases related to the abnormal expression of CKIP-1 gene. The small interfering nucleic acid is capable of cross-species inhibiting the CKIP-1 gene expression, inhibiting CKIP-1 expression in human, rhesus, rats and mice simultaneously, and facilitating the differentiation of osteoblasts and mineralization of bone matrix effectively.

Abstract: The present invention provides siRNAs for inhibiting the expression of plk1 gene, and the method for inhibiting the expression of plk1 gene in mammalian cells. The siRNAs of the present invention have the double-stranded structure, and said double-stranded structure is composed of the first single strand and the second single strand that are fully complementary, wherein the sequence of said first single strand is the same as the target sequence within the sequence as shown in SEQ ID NO: 1, and the sequence of said second single strand is complementary to the target sequence within the sequence as shown in SEQ ID NO: 1. The siRNAs of the present invention can sequence specifically mediate the inhibition of plk1 gene expression, and have a good serum stability. By the introduction of the siRNAs of the present invention into the tumor cells, the expression of plk1 gene can be effectively inhibited, and the growth of tumor cells is inhibited and the apoptosis of tumor cells is promoted.

Abstract: Provided are a polycaprolactone-polyphosphate block copolymer, a liquid composite formed by the block copolymer, a nucleic acid preparation, preparation methods for the copolymer and the liquid composite, and the use of the copolymer and the liquid composite in a nucleic acid medicine delivery system. The block copolymer prepared using the present invention has good biocompatibility, low cytotoxicity, and good biodegradability. The micelles provided in the present invention self-assemble into nano-particles in an aqueous solution, and have good stability, biocompatibility, and biodegradability, and low cytotoxicity. The preparation method is simple, has high repeatability, as a vector can protect small nucleic acids such as siRNA from biodegradation, can combine with the scale effect of nano-particles, and can be used for treating different diseases. Additionally, bonding targeting groups enable specificity recognition of different cancer cells.

Abstract: Provided are a double-stranded nucleic acid and use thereof in ribonuclease detection, and a ribonuclease detection method and use thereof in tumor detection and/or diagnosis. Specifically, a double-stranded nucleic acid substrate comprises at least one ribonuclease sensitive site. The activity and content of the ribonuclease in a sample are detected by analyzing the degradation of the double-stranded nucleic acid substrate by the ribonuclease. Also provided are a ribonuclease detection kit and a tumor detection kit.

Abstract: A method for preparing oligonucleotide comprising reacting the compound of Formula (1) with the compound of Formula (2) in a liquid reaction medium under the condition of condensation reaction to obtain the compound of formula (3) is provided. 1-(2-mesitylenesulfonyl)-3-nitro-1H-1,2,4-triazole (MSNT) is applied as condensing agent. Oligonucleotides synthesized in the liquid reaction medium could be obtained on a large scale.

Abstract: Nucleotide and/or oligonucleotide represented by formula (1) and the liquid phase synthesis process thereof. The present invention provides a liquid phase synthesis process for preparing a nucleotide and/or an oligonucleotide, comprising a process for combining the nucleotide and/or oligonucleotide protective groups, in which, under the condition that the 2?-hydroxyl group is protected by a group with a sterically hindered silane structure, the 3? phosphate group(s) of the nucleotide and/or oligonucleotide is/are directly protected by (a)?-cyanoethyl group(s), and after the ?-cyanoethyl group(s) is/are removed, the resulting product can directly participate in the next cycle of synthesis, wherein the synthesis reaction is carried out in a reaction flask or reaction kettle, without being limited by a solid carrier or synthesizer, so that the large scale preparation of oligonucleotides can be achieved.

Abstract: Inhibitors that can inhibit expression of FAM3B gene to reduce the levels of expression products, or can combine the expression products to reduce the activity of promoting lipid synthesis of FAM3B gene product are provided, wherein the inhibitors are one or more inhibitors selected from the group consisting of small interfering RNAs, antisense oligonucleotides, antibodies against FAM3B proteins and active organic compounds. Cells, vectors or inhibitor compositions, comprising such inhibitors, methods for inhibiting expression of FAM3B gene or inhibiting the activity of promoting lipid synthesis of FAM3B gene product using the inhibitors are provided. Methods for treating diseases mediated by expression of FAM3B gene using such inhibitors and uses of the inhibitors in preparing pharmaceuticals for preventing and/or treating the disease mediated by FAM3B gene expression are also provided.

Abstract: A method for preparing oligonucleotide comprising reacting the compound of Formula (1) with the compound of Formula (2) in a liquid reaction medium under the condition of condensation reaction to obtain the compound of formula (3). In the method according to the present invention, the functional groups are protected by suitable protective groups to only expose the 5?-OH of the compound of Formula (1) (OH-component) and the 3?-phosphate of the compound of Formula (2) (P-component) which are to be connected, so that the condensation reaction is carried out in a liquid reaction medium to bond the OH-component and P-component to obtain DNA or RNA short chain. The method of the present invention does not need a solid phase column and can be carried out in a liquid reaction medium. Thus, oligonucleotides can be synthesized on a large scale.

Abstract: The present invention provides a complex molecule interfering the expression of target genes and the methods for preparing the complex molecule, wherein the complex molecule contains two siRNA strands X1 and X2 having at least 80% complementarity, the 5? end of X1 and 3? end of X2 are linked through non-nucleic acid molecule L1, the 5? end of X2 and 3? end of X1 are linked through non-nucleic acid molecule L2. Since both 5? and 3? ends of two siRNA strands X1 and X2 of the complex molecule according to the present invention are linked through non-nucleic acid molecules, it is not easy to unwind and degraded for the siRNA strands, and therefore the chemical stability of siRNA and the remaining time in the blood are greatly improved. After being administered, the Dicer enzyme in the cells is utilized to release the locked siRNAs from the complex molecules, and after unwinding, the antisense strand of the siRNA is released from the double-stranded siRNA to inhibit the expression of the target genes.