Abstract: RNAi agents (e.g., CYP7A1 RNAi agents) for inhibiting the expression of the CYP7A1 gene, compositions including the RNAi agents conjugated to a targeting moiety, and methods of use are described. Also disclosed are pharmaceutical compositions including one or more RNAi agents (e.g., CYP7A1 RNAi agents). Delivery of the RNAi agent(s) to liver cells in vivo inhibits CYP7A1 gene expression and treats a CYP7A1 disease or a CYP7A1-associated disease.

Abstract: The present invention provides oligonucleotides comprising 2?-O-methyl (2?-OMe) and 2?-deoxy-2?-fluoro (2?-F) modifications, compositions thereof, and methods of use for reducing the expression or activity of a gene.

Abstract: RNAi agents (e.g., CYP7A1 RNAi agents) for inhibiting the expression of the CYP7A1 gene, compositions including the RNAi agents conjugated to a targeting moiety, and methods of use are described. Also disclosed are pharmaceutical compositions including one or more RNAi agents (e.g., CYP7A1 RNAi agents). Delivery of the RNAi agent(s) to liver cells in vivo inhibits CYP7A1 gene expression and treats a CYP7A1 disease or a CYP7A1-associated disease.

Abstract: Provided herein are double-stranded nucleic acid inhibitor molecules having a shortened sense strand with a stem loop structure and an antisense strand. Also provided are methods and compositions for reducing target gene expression and methods and compositions for treating a disease of interest.

Abstract: RNAi agents (e.g., CYP7A1 RNAi agents) for inhibiting the expression of the CYP7A1 gene, compositions including the RNAi agents conjugated to a targeting moiety, and methods of use are described. Also disclosed are pharmaceutical compositions including one or more RNAi agents (e.g., CYP7A1 RNAi agents). Delivery of the RNAi agent(s) to liver cells in vivo inhibits CYP7A1 gene expression and treats a CYP7A1 disease or a CYP7A1-associated disease.

Abstract: This disclosure relates to oligonucleotides, compositions and methods useful for reducing LDHA expression, particularly in hepatocytes.

Abstract: Disclosed herein are oligonucleotides, such as nucleic acid inhibitor molecules, having a 4?-phosphate analog and methods of using the same, for example, to modulate the expression of a target gene in a cell. The phosphate analogs are bound to the 4?-carbon of the sugar moiety (e.g., a ribose or deoxyribose or analog thereof) of the 5?-terminal nucleotide of an oligonucleotide. Typically, the phosphate analog is an oxymethylphosphonate, where the oxygen atom of the oxymethyl group is bound to the 4?-carbon of the sugar moiety or analog thereof.

Abstract: This disclosure relates to oligonucleotides, compositions and methods useful for reducing LDHA expression, particularly in hepatocytes.

Abstract: Provided herein are double-stranded nucleic acid inhibitor molecules having a sense strand with a stem loop structure and an antisense strand, where the stem portion of the stem loop structure contains one or more Tm-increasing nucleotides. Also provided are methods and compositions for reducing target gene expression and methods and compositions for treating a disease of interest.

Abstract: This disclosure relates to oligonucleotides, compositions and methods useful for reducing LDHA expression, particularly in hepatocytes.

Abstract: Disclosed herein are oligonucleotides, such as nucleic acid inhibitor molecules, having a 4?-phosphate analog and methods of using the same, for example, to modulate the expression of a target gene in a cell. The phosphate analogs are bound to the 4?-carbon of the sugar moiety (e.g., a ribose or deoxyribose or analog thereof) of the 5?-terminal nucleotide of an oligonucleotide. Typically, the phosphate analog is an oxymethylphosphonate, where the oxygen atom of the oxymethyl group is bound to the 4?-carbon of the sugar moiety or analog thereof.

Abstract: The present invention provides oligonucleotides comprising 2?-O-methyl (2?-OMe) and 2?-deoxy-2?-fluoro (2?-F) modifications, compositions thereof, and methods of use for reducing the expression or activity of a gene.

Abstract: The present invention relates to methods for synthesizing compounds useful as potent and stable RNA interference agents, derivatives thereof, and intermediates thereto.

Abstract: Disclosed herein are oligonucleotides, such as nucleic acid inhibitor molecules, having a 4?-phosphate analog and methods of using the same, for example, to modulate the expression of a target gene in a cell. The oligonucleotide of the disclosure comprises a 5?-terminal nucleotide represented by Formula III: wherein Ra, Rb, B, X2 and Y are as defined in the specification. The phosphate analogs are bound to the 4?-carbon of the sugar moiety (e.g., a ribose or deoxyribose or analog thereof) of the 5?-terminal nucleotide of an oligonucleotide. Typically, the phosphate analog is an oxymethylphosphonate, where the oxygen atom of the oxymethyl group is bound to the 4?-carbon of the sugar moiety or analog thereof.

Abstract: Provided herein are double-stranded nucleic acid inhibitor molecules having a shortened sense strand with a stem loop structure and an antisense strand. Also provided are methods and compositions for reducing target gene expression and methods and compositions for treating a disease of interest.

Abstract: This disclosure relates to oligonucleotides, compositions and methods useful for reducing LDHA expression, particularly in hepatocytes.

Abstract: This disclosure relates to oligonucleotides, compositions and methods useful for reducing LDHA expression, particularly in hepatocytes.

Abstract: This disclosure relates to oligonucleotides, compositions and methods useful for reducing LDHA expression, particularly in hepatocytes.

Abstract: Provided herein are double-stranded nucleic acid inhibitor molecules having a sense strand with a stem loop structure and an antisense strand, where the stem portion of the stem loop structure contains one or more Tm-increasing nucleotides. Also provided are methods and compositions for reducing target gene expression and methods and compositions for treating a disease of interest.

Abstract: Disclosed herein are oligonucleotides, such as nucleic acid inhibitor molecules, having a 4?-phosphate analog and methods of using the same, for example, to modulate the expression of a target gene in a cell. The phosphate analogs are bound to the 4?-carbon of the sugar moiety (e.g., a ribose or deoxyribose or analog thereof) of the 5?-terminal nucleotide of an oligonucleotide. Typically, the phosphate analog is an oxymethylphosphonate, where the oxygen atom of the oxymethyl group is bound to the 4?-carbon of the sugar moiety or analog thereof.