Provided herein are methods, compounds, and compositions for reducing expression of GYS1 in an individual. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a glycogen storage disease or disorder in an individual in need.

The present invention provides an amidite compound capable of improving a yield and a purity of a polyoligonucleotide, a glycoside compound as an intermediate thereof, and a production method for a polynucleotide using the amidite compound. The present invention also provides an amidite compound of formula (1) capable of improving a yield and a purity of a polyoligonucleotide, a glycoside compound of formula (10) (in formulae (10) and (1), B.sup.a, R.sup.a, R.sup.b, R.sup.c, G.sup.1, G.sup.2, and G.sup.3 are as defined in the description, and R is represented by the following formulae), and a production method for a polynucleotide using the amidite compound.

Provided is a method of treating cancer cells localized in the lung by administering to such patients a therapeutically effective amount of a liposomal annamycin formulation (L-Ann).

Methods of labelling one or more subcellular components (e.g., an organelle and/or subcellular region) in vivo are provided. Methods of labelling a protein in vivo are provided. Methods of determining a nucleic acid sequence in situ are also provided.

Methods of labelling one or more subcellular components (e.g., an organelle and/or subcellular region) in vivo are provided. Methods of labelling a protein in vivo are provided. Methods of determining a nucleic acid sequence in situ are also provided.

A sample containing particles having high-molecular-weight (HMW) DNA is entrapped in a gel matrix, and the gel matrix is exposed to a lysis reagent configured to release the HMW DNA from the particles. The HMW DNA may be purified by subjecting the gel matrix to an electrophoretic field that removes the HMW DNA from the particles, lysis reagents, and/or other sample constituents, from the gel matrix such that the HMW DNA remains. The gel matrix may be subjected with DNA cleavase re-agents configured to cleave at specific DNA sequences within the HMW DNA to liberate defined segments of the DNA as fragments of reduced size. The gel matrix may also be subjected to an electrophoretic field, which moves and separates the DNA fragments from uncleaved DNA of the HMW DNA, which remains substantially immobile. The electrophoretically separated DNA fragments may be isolated from the gel matrix.

The present invention includes synthesis of polyethyleneimine800-EpoxyC8-22 (PEI800-C8-22) lipids, e.g., Polyethyleneimine800-EpoxyC16 (PEI800-C16), PEI12C16, PEI8C16, and PEI4C16 lipids, compositions and methods for transfecting primary leukocytes, myeloid cells, lymphoid cells, monocytes, macrophages and dendritic cells (DC) comprising a transfection complex comprising: one or more nanoparticles; and Polyethyleneimine800-EpoxyC16 (PEI800-C16), PEI12C16, PEI8C16, and PEI4C16 lipids complexed with one or more nucleic acids, such as, e.g., DNA, RNA, nucleic acid vectors, shRNA, miRNA, and RNAi on or about the nanoparticles.

Described herein are DNA-nanostructures that can be used in an assay to detect and/or quantify an analyte of interest. Aspects of the DNA-nanostructure can include a single DNA molecule composed of hairpin structural motifs, an anchor recognition moiety, and a signal moiety, where the anchor recognition moiety and the signal moiety are in effective proximity to each other such that the tethered diffusion of the signal molecule can be altered based upon binding status of the anchor recognition moiety. Also described herein are methods of making and using the DNA-nanostructures.

The compositions and methods of the invention provide compositions and methods for preferential targeting of tissues to delivery therapeutic or diagnostic agents. For example, such compounds are useful in the treatment of joint disorders those affecting articulating joints, e.g., injury-induced osteoarthritis as well as autoimmune diseases affecting joint tissue such as rheumatoid arthritis.

The present invention relates to compounds of formula (I), their diastereoisomers and their salts, for use for inhibiting cancer-related inflammation, notably via STING interaction.

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