Disclosed herein are antisense compounds and methods for decreasing LDL-C in an individual having elevated LDL-C. Additionally disclosed are antisense compounds and methods for treating, preventing, or ameliorating hypercholesterolemia and/or atherosclerosis. Further disclosed are antisense compounds and methods for decreasing coronary heart disease risk. Such methods include administering to an individual in need of treatment an antisense compound targeted to a PCSK9 nucleic acid. The antisense compounds administered include gapmer antisense oligonucleotides.

Provided are methods relating to SIRPgamma as a biomarker for cancer cells, and in particular of cancer stem cells and lung adenocarcinoma. Disclosed are also methods of treatment treatment, which involve administering a SIRPgamma targeted agent to a subject with cancer, alone or with another cancer therapy. Methods for diagnosing cancer, identifying subjects with high SIRPgamma levels for treatment, and monitoring SIRPgamma expressing tumors are also provided.

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an alpha-2A adrenergic receptor (ADRA2A) gene, as well as methods of inhibiting expression of an ADRA2A gene and methods of treating subjects having an ADRA2A-associated disease or disorder, e.g., a primary tauopathy or Alzheimer's disease, using such dsRNAi agents and compositions.

The present invention provides a method of treating a nucleotide repeat expansion disorder comprising delivering a pair of engineered nucleases, or genes encoding engineered nucleases, to the cells of a patient such that the two nucleases excise the nucleotide repeat responsible for the disease permanently from the genome. The invention provides a general method for treating nucleotide repeat expansion disorders and engineered nucleases suitable for practicing the method. The invention further provides vectors and techniques for delivering engineered nucleases to patient cells.

Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described.

The present invention relates to methods for the identification of anti-HIV miRNAs and anti-HIV pharmaceutical compounds using high-throughput screening methods, comprising: transfecting reporter cells with a panel of miRNAs, infecting the reporter cells with HIV, screening the cells to identify miRNAs that modulate HIV infection and identifying the specific pathways, nucleic acids and/or polypeptides that are targeted by the miRNAs. The invention further provides for the identification and screening of anti-HIV pharmaceutical compounds having known activity against the specific pathways, nucleic acids and/or polypeptides that are targeted by the miRNAs for efficacy in the treatment of HIV. The invention also provides for the use of miRNA mimics, miRNA inhibitors and pharmaceutical compounds (including oncology drugs and kinase inhibitors) in the treatment and/or prevention of HIV infection.

Methods of accelerating or improving the healing of a skin graft or skin grafting site in a subject are provided comprising administering to the subject an amount of an inhibitor of fidgetin-like 2.

The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs (siRNAs) that selectively silence targeted genes (by cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.

The invention provides methods of detecting cancer biomarkers, such as one or more SWI/SNF complex mutations, in order to determine a cancer subject's amenability to therapeutic treatment with a BRM inhibitor. Kits, methods of screening for candidate BRM inhibitors, and associated methods of treatment are also provided.

The present invention provides an aptamer that binds to IL-17 to inhibit binding of IL-17 and IL-17 receptor; a complex containing the aptamer and a functional substance (e.g., affinity substance, labeling substance, enzyme, drug delivery medium, drug and the like); a medicament containing the aptamer, or a complex containing the aptamer and a functional substance, a diagnosing drug and labeling agent and the like.