Abstract: A method for reducing neovascularization in an ocular tissue is carried out by contacting the tissue with an inhibitor of endomucin expression or activity.

Abstract: The present invention provides an antisense nucleic acid medicine that can modulate expression of a target transcriptional product in an ischemic site of a subject. The present invention also provides a composition for modulating expression of a target transcriptional product in an ischemic site of a subject, having a nucleic acid complex formed by annealing together a first nucleic acid strand having an antisense oligonucleotide region with respect to the target transcriptional product, and a lipid-conjugated second nucleic acid strand having a complementary region that is complementary to at least part of the first nucleic acid strand.

Abstract: The present invention relates to methods for performing antisense oligonucleotide-mediated exon skipping in the retina of a subject in need thereof. In particular, the present invention relates to a method for performing antisense oligonucleotide-mediated exon skipping in a retina cell of a subject comprising the step of injecting into the vitreous of the subject an amount of the antisense oligonucleotide.

Abstract: Disclosed are nanostructures comprising a ribonucleic acid (RNA) scaffold comprising a hexameric tetrahedral core. The tetrahedral core may comprise four hexameric RNA nanorings linked together. Related pharmaceutical compositions, methods of modulating the expression of a target gene in a mammal, methods of treating or preventing a disease in a mammal, and methods of producing a hexameric tetrahedral RNA nanostructure are also disclosed.

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

Abstract: The present disclosure relates to compositions and methods for determining the quality of stored blood and detecting Autologous Blood Transfusions (ABT) and blood doping.

Abstract: Disclosed herein are conditional siRNAs activatable by CBF?-MYH11 oncogenic gene and use thereof for treating conditions such as acute myeloid leukemia (AML). The conditional siRNAs target MCL-1 or HDAC8.

Abstract: A compound for forming a conjugate with an active agent such as an oligonucleotide having a structure represented by Formula (321). The present disclosure also provides a corresponding conjugate. The conjugate of the present disclosure can specifically target hepatocytes, thereby effectively solve the problems associated with delivery of oligonucleotide drugs in vivo, and have low toxicity and excellent delivery efficiency while maintaining high stability for the delivered oligonucleotide.

Abstract: A cardiovascular graft is provided, such as a prosthetic heart valve or blood vessel, that comprises mesenchymal stem cells (MSCs) and/or progeny thereof, modified to knock down or knockout expression of the telomerases reverse transcriptase gene (TERT), or otherwise reduce activity of TERT. Also provided are a method of making the cardiovascular graft, and a method of implanting the cardiovascular graft.

Abstract: Provided herein are, inter alia, nucleic acid-peptide conjugates including a non-cell penetrating protein (e.g., an Hdm2 targeting peptide) attached at its C-terminus to a phosphorothioate nucleic acid. Attachment of the phosphorothioate nucleic acid to the non-cell penetrating protein conveys stability to and allows for efficient intracellular delivery of the non-cell penetrating peptide. The nucleic acid-peptide conjugates provided herein including embodiments thereof are useful, inter alia, for the treatment of cancer.

Abstract: The present disclosure provides, in part, methods of discovering immunotherapy targets in vivo, therapeutic compositions (e.g., shRNA, immunoresponsive cells expressing shRNA and/or a chimeric antigen receptors (CAR)), and methods of use thereof.

Abstract: The present disclosure provides antisense oligonucleotides, compositions, and methods that target ATP7B exon 6 or a flanking intron, thereby modulating splicing of ATP7B pre-mRNA to increase the level of ATP7B mRNA molecules having exon 6, e.g., to provide a therapy for Wilson disease. The present disclosure provides an antisense oligonucleotide including a nucleobase sequence at least 70% complementary to an ATP7B target sequence in exon 6, a 5?-flanking intron, a 3?-flanking intron, or a combination of exon 6 and the 5?-flanking or 3?-flanking intron.

Abstract: The present disclosure provides method of preventing, lessening the effects, or treating cytokine release syndrome (CRS) or related disorders, and/or neurotoxicity associated with immunotherapy comprising administering defibrotide. The defibrotide can be administered after the immunotherapy begins or be administered prophylactically before immunotherapy begins or before the patient develops CRS and/or neurotoxicity.

Abstract: The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.

Abstract: The present invention provides compositions and methods for the treatment or prevention of a lysosomal disease or disorder involving increasing the level, expression, or activity of a metallothionein polypeptide or polynucleotide in the subject.

Abstract: The disclosure provides a composition for suppressing inflammation comprising at least one substance that disrupts a stem-loop structure in the 3? untranslated region of a Regnase-1 mRNA, wherein the stem-loop structure is at least one stem-loop structure selected from a first stem-loop structure formed in a region corresponding to positions 231 to 245 of SEQ ID NO: 1 and a second stem-loop structure formed in a region corresponding to positions 424 to 442 of SEQ ID NO: 1.

Abstract: This disclosure relates to RNA interference (RNAi) reagents for treatment of hepatitis B virus (HBV) infection, compositions comprising same, and use thereof to treat individuals infected with HBV. The reagents are artificial miRNA (shmiRNA) used alone or in combination with additional shmiRNA or shRNA.

Abstract: Provided herein, inter alia, are double stranded oligonucleotide molecules and methods of making the molecules. The double stranded oligonucleotide molecules include a first oligonucleotide strand comprising a first nucleic acid sequence bound to a second nucleic acid sequence through a first spacer, wherein said second nucleic acid sequence is bound to a third nucleic acid sequence through a second spacer and a second oligonucleotide strand comprising a fourth nucleic acid sequence bound to a fifth nucleic acid sequence through a third spacer, wherein said fifth nucleic acid sequence is bound to a sixth nucleic acid sequence through a fourth spacer, wherein the second nucleic acid sequence and the fifth nucleic acid sequence are hybridized to form a double stranded nucleic acid core of said double stranded oligonucleotide.

Abstract: 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.

Abstract: The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 400 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in direction selective retinal ganglion cells of a gene when operatively linked to a nucleic acid sequence coding for said gene.