Abstract: Methods of treating Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), comprising administering an inhibitory nucleic acid that targets miR-128.

Abstract: Disclosed are oligonucleotides, compositions, and methods that may be useful in the treatment of age-related macular degeneration (AMD). The treatment of age-regulated macular degeneration (AMD) may involve inhibiting an miR-33 target nucleic acid. For example, inhibition of an miR-33 target nucleic acid may be achieved using antisense oligonucleotides targeting an miR-33 target nucleic acid, interfering oligonucleotides targeting an miR-33 target nucleic acid, or recombinant AAV particles including a vector encoding an antisense oligonucleotide or interfering oligonucleotide targeting an miR-33 target nucleic acid.

Abstract: It has been discovered that NAD+-dependent histone deacetylase SIRT6 is critical for suppression of PDAC by controlling the expression of Lin28b, which is a negative regulator of let-7 microRNA. Specifically, SIRT6 loss results in histone hyperacetylation at the Lin28b promoter, Myc recruitment, and pronounced induction of Lin28b and downstream let-7 target genes, HMGA2, IGF2BP1, and IGF2BP3. This invention relates generally to agents and methods of reducing expression or activity of Lin28b to treat (aggressive) PDAC in a subject.

Abstract: Methods of treating Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), comprising administering an inhibitory nucleic acid that targets miR-128.

Abstract: It has been discovered that NAD+-dependent histone deacetylase SIRT6 is critical for suppression of PDAC by controlling the expression of Lin28b, which is a negative regulator of let-7 microRNA. Specifically, SIRT6 loss results in histone hyperacetylation at the Lin28b promoter, Myc recruitment, and pronounced induction of Lin28b and downstream let-7 target genes, HMGA2, IGF2BP1, and IGF2BP3. This invention relates generally to agents and methods of reducing expression or activity of Lin28b to treat (aggressive) PDAC in a subject.

Abstract: Methods of treating Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), comprising administering an inhibitory nucleic acid that targets miR-128.

Abstract: Methods of treating Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), comprising administering an inhibitory nucleic acid that targets miR-128.

Abstract: It has been discovered that NAD+-dependent histone deacetylase SIRT6 is critical for suppression of PDAC by controlling the expression of Lin28b, which is a negative regulator of let-7 microRNA. Specifically, SIRT6 loss results in histone hyperacetylation at the Lin28b promoter, Myc recruitment, and pronounced induction of Lin28b and downstream let-7 target genes, HMGA2, IGF2BP1, and IGF2BP3. This invention relates generally to agents and methods of reducing expression or activity of Lin28b to treat (aggressive) PDAC in a subject.

Abstract: Methods for treating cancer, e.g., cancer of epithelial origin, by specifically targeting human satellite II (HSATII) using sequence specific agents such as oligonucleotides.

Abstract: Methods of treating Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), comprising administering an inhibitory nucleic acid that targets miR-128.

Abstract: Methods for treating cancer, e.g., cancer of epithelial origin, by specifically targeting human satellite II (HSATII) using sequence specific agents such as oligonucleotides. As shown herein, the hetero chromatic HSATII satellite repeat is silenced in normal cells, but massively over expressed in epithelial cancers and in cancer cell lines when grown as xenografts or in 3D culture. Induction of HSATII RNA, either in xenografts or using in vitro reconstitution models, suggests the appearance of complementary DNA intermediates.

Abstract: Compositions comprising nucleic acid sequences that target MiR-33 microRNAs are described, together with uses of the same in the treatment of cholesterol-related disorders.

Abstract: It has been discovered that NAD+-dependent histone deacetylase SIRT6 is critical for suppression of PDAC by con trolling the expression of Lin28b, which is a negative regulator of let-7 microRNA. Specifically, SIRT6 loss results in histone hyperacetylation at the Lin28b promoter, Myc recruitment, and pronounced induction of Lin28b and downstream let-7 target genes, HMGA2, IGF2BP1, and IGF2BP3. This invention relates generally to agents and methods of reducing expression or activity of Lin28b to treat (aggressive) PDAC in a subject.

Abstract: Methods for targeting microRNA 128 (miR-128) for regulating cholesterol/lipid metabolism and insulin sensitivity.

Abstract: Compositions comprising nucleic acid sequences that target MiR-33 microRNAs are described, together with uses of the same in the treatment of cholesterol-related disorders.

Abstract: Methods for treating cancer, e.g., cancer of epithelial origin, by specifically targeting human satellite II (HSATII) using sequence specific agents such as oligonucleotides. As shown herein, the hetero chromatic HSATII satellite repeat is silenced in normal cells, but massively over expressed in epithelial cancers and in cancer cell lines when grown as xenografts or in 3D culture. Induction of HSATII RNA, either in xenografts or using in vitro reconstitution models, suggests the appearance of complementary DNA intermediates.

Abstract: Methods for targeting microRNA 128 (miR-128) for regulating cholesterol/lipid metabolism and insulin sensitivity.

Abstract: Methods for targeting microRNA 128 (miR-128) for regulating cholesterol/lipid metabolism and insulin sensitivity.

Abstract: Elevated blood levels of low-density lipoprotein-cholesterol (LDL-C, or “bad” cholesterol) are strongly linked to circulatory disorders, e.g. cardiovascular disease such as atherosclerosis, angina, coronary heart disease, heart attack, stroke, etc. The LDL receptor (LDLR) mediates uptake of LDL-C (low density lipoprotein-cholesterol) by, e.g. hepatic cells. As described herein, miR-148a regulates LDLR expression in human hepatic cells. Accordingly, described herein are methods and compositions relating to, e.g. regulating cholesterol levels by modulating the level of miR-148a.

Abstract: Methods for targeting microRNA 128 (miR-128) for regulating cholesterol/lipid metabolism and insulin sensitivity.