Abstract: The present invention relates to ligand conjugates of oligonucleotides (e.g., iRNA agents) and methods for their preparation. The ligands are derived primarily from monosaccharides These conjugates are useful for the in vivo delivery of oligonucleotides.

Abstract: The present embodiments provide compounds and methods for targeting cells expressing GLP-1 receptor.

Abstract: The present invention relates to the field of genetic engineering, in particular, to a transposon-based transfection kit suitable for transfection of primary cells, such as T cells, comprising mRNA encoding a transposase, or reagents for generating mRNA encoding said transposase, as well as minicircle DNA comprising the transposon. The invention also relates to a nucleic acid, preferably, a DNA minicircle, comprising a transposon, wherein the transposon encodes a protein and at least one miRNA, wherein the sequences encoding the miRNA are located in an intron and expression of the protein and the miRNA is regulated by the same promoter. The invention also provides a population of cells obtainable with the method of the invention. Methods of transfection are also provided, as well as medical use, e.g. in immunotherapy, in particular, in adoptive T cell therapy or T cell receptor (TCR) or chimeric antigen receptor (CAR) gene therapy.

Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a suspension of preformed lipid nanoparticles and mRNA.

Abstract: Provided are methods and compositions for activating oligonucleotide aptamer-deactivated DNA polymerases, comprising cleaving the aptamer by endonuclease V enzymatic activity to reduce or eliminate binding of the oligonucleotide aptamer to the DNA polymerase, thereby activating DNA synthesis activity of the DNA polymerase in a reaction mixture. Mixtures for use in methods of the invention are also provided. The oligonucleotide aptamers of the present invention are circular and comprise one or more deoxyinosine nucleotides providing for aptamer-specific recognition and cleavage of the circular aptamer by the endonuclease V enzymatic activity. Exemplary oligonucleotide aptamers, mixtures and methods employing endonuclease V enzymatic activity are provided.

Abstract: The present disclosure relates to genetically modified arthropods, genetically modified bacteria, and methods for controlling and/or reducing arthropod populations.

Abstract: The presently-disclosed subject matter relates to an artificial RNA nanostructure and method of use thereof. In particular, the presently-disclosed subject matter relates to RNA nanoparticles and RNA dendrimers, and methods of disease diagnosis and treatments using the RNA nanostructure and RNA dendrimers.

Abstract: Provided herein are aptamers capable of inhibiting the activity of Von Willebrand Factor (VWF). Pharmaceutical compositions comprising these aptamers are also provided. Methods of preventing blood clot formation in a subject by administering the aptamers are provided and methods of treating a blood clot by administering a VWF-targeting agent are also provided.

Abstract: Provided are hybrid tRNA/pre-miRNA molecules, e.g., comprising a single tRNA and one, two or more pre-miRNA molecules, useful for the production and therapeutic delivery of an inserted RNA sequence, e.g., one or more miRNAs. Also provided are liposomes and nanoparticles that include the hybrid tRNA/pre-miRNA molecules. Methods of treating cancer by administration of the hybrid tRNA/pre-miRNA molecules are also provided.

Abstract: The present invention relates to a pharmaceutical composition for preventing or treating hypertrophic scars. The present inventors have found that the inhibition of expression of TXNDC5, PRRC1, S100A11, Galectin 1, Filamin A, eIF-5A, Annexin A2, and FABP5 can be a new target for improving and treating hypertrophic scars. In the present invention, TXNDC5-, PRRC1-, S100A11-, Galectin 1-, Filamin A-, eIF-5A-, Annexin A2-, and FABP5-specific siRNAs were constructed to determine the probability of treating the hypertrophic scars. As a result, the knockdown of the protein or a gene encoding the protein induces apoptosis in the hypertrophic scars and reduces collagen expression, which can be very useful in treating wounds.

Abstract: The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.

Abstract: Described herein are compositions that inhibit/degrade STAT3, and methods of using such compositions for chemoprevention of non-small cell lung cancer (NSCLC).

Abstract: The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.

Abstract: The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).

Abstract: Methods for increasing specificity of RNA-guided genome editing, e.g., editing using CRISPR/Cas9 systems.

Abstract: Disclosed are nucleic acid oligomer compounds and to their use in compositions and methods for inhibiting proliferation, survival or viability of cancer cells including prostate, lung, pancreatic, breast, cervical and bone cancer cells.

Abstract: The present disclosure relates to antisense oligomers and related compositions and methods for increasing the expression of functional human type VII collagen and methods for treating dystrophic epidermolysis bullosa and related disorders and relates to inducing exclusion of exon 80 in human type VII collagen mRNA.

Abstract: An object of the present invention is to develop a novel treatment method for chronic diseases for which conventional treatment methods are either ineffective or for which efficacy is low. The present invention provides a pharmaceutical composition for the treatment and/or prevention of an inflammatory chronic disease that is used in combination with a biological preparation that inhibits leukocyte tissue invasion. The pharmaceutical composition of the present invention contains as an active ingredient thereof siRNA suppressing the expression of CHST15 gene that contains a structure formed by the hybridization of RNA containing the base sequence represented by SEQ ID NO: 1 with RNA complementary thereto.

Abstract: The present invention aims to provide a nucleic acid compound that hardly forms non-Watson-Crick base pairs, and an oligonucleotide containing the nucleic acid compound and showing reduced non-specific binding with nucleic acids other than the target nucleic acid. The nucleic acid compound according to the present invention is characterized in that the 2-position carbonyl group of the pyrimidine base is functionally converted (X1 and X2 are each independently S or Se), and that the 2?-position and the 4?-position are bridged in a particular structure. The oligonucleotide according to the present invention is characterized in that at least one of thymidine and uridine is the nucleic acid compound.

Abstract: The present invention discloses a combinational therapy for enhancing efficacy of immune checkpoint blockade for tumors with immune suppressive microenvironment. More specifically, this combination therapy involves the treatment of cancer through immune checkpoint inhibitors and CpG-oligodeoxynucleotides.