Abstract: Modified release formulations of viloxazine and methods of administering the same are disclosed. High-drug load formulations of viloxazine are further disclosed.

Abstract: Seven protective alleles for IgA nephropathy have been discovered that can be identified by analyzing a DNA sample for seven respective SNPs. A method is provided for identifying and treating subjects at risk of developing IgA neuropathy based on a new seven-SNP genetic risk score. Also provided are screening methods to identify compounds that bind to and reduce the expression or biological activity of a either CFHR1 or CFHR3.

Abstract: The invention provides transdermal delivery systems, medical kits, and methods for using the transdermal delivery systems and kits for medical applications, such as delivery of contraceptive agents to control fertility.

Abstract: This invention provides methods and compositions for preventing, treating or ameliorating one or more symptoms of a malignant tumor associated with KRAS mutation in a mammal in need thereof, by identifying a tumor cell in the mammal, the tumor cell comprising at least one of: (i) a mutation of the KRAS gene, and (ii) an aberrant expression level of KRAS protein; and administering to the mammal a therapeutically effective amount of a composition comprising one or more RNAi molecules that are active in reducing expression of GST-?.

Abstract: Compositions and methods for enhancing delivery of molecules, e.g. biological agents, into cells are described. The composition is a conjugate of the biological agent, preferably a nucleic acid analog having a substantially uncharged backbone, covalently linked to a peptide transporter moiety as described. Conjugation of the peptide transporter to a substantially uncharged nucleic acid analog, such as a morpholino oligomer, is also shown to enhance binding of the oligomer to its target sequence and enhance antisense activity.

Abstract: A method of generating a particle is disclosed, the particle being for delivery of a polynucleotide to a target cell. The method comprises (a) contacting the polynucleotide with a composition comprising cationic molecules, wherein the cationic molecules condense the polynucleotide by electrostatic interactions to generate a complex, wherein the cationic molecules are not comprised in a liposome; and (b) covalently binding the complex to a targeting moiety at a pH equal to or below about 4.5, thereby generating the particle for delivery of the polynucleotide agent to the target cell. Use of the particles and compositions comprising same are also disclosed.

Abstract: Innovative graft polymers designed for the efficient delivery of antisense molecules into biological cells and for maintaining the biological activity of these molecules while in serum and other aqueous environments are provided. Such polymers may comprise an anionic graft polymer comprising an anionic polymer backbone with pendant carboxylic acid groups and pendant chains comprising amphipathic or hydrophilic polymers covalently bonded to a portion of said pendant carboxylic acid groups. Antisense molecule delivery vectors comprising such polymers in combination with cationic agents for delivery of antisense molecules are also disclosed.

Abstract: A therapeutic agent for a corneal epithelial disorder, containing a miR-203 inhibitor; an agent for promoting proliferation of corneal epithelial cells, containing a miR-203 inhibitor; a liquid culture medium for use in the manufacture of a corneal epithelial sheet, containing a miR-203 inhibitor; and a method for producing a corneal epithelial sheet, including the step of culturing corneal epithelial cells using a liquid culture medium containing a miR-203 inhibitor. According to the present invention, a therapeutic agent for a corneal epithelial disorder, an agent for promoting proliferation of corneal epithelial cells, a liquid culture medium for use in the manufacture of a corneal epithelial sheet, a method for producing a corneal epithelial sheet, including the step of culturing corneal epithelial cells using the liquid culture medium, and a method for treating a corneal epithelial disorder can be provided.

Abstract: A compound composed of a phospholipid and basic amino acid residues is provided as is a sterically stabilized phospholipid nanocarrier containing the compound and use of the same in passive and targeted delivery of negatively charged therapeutic agents encapsulated within the nanocarrier.

Abstract: Described are methods for early diagnosis and progression monitoring of Mild Cognitive Impairment (MCI) and Alzheimer's Disease (AD) by quantifying neurite and/or synapse miRNAs in bodily fluids.

Abstract: This invention relates to the inhibition of expression or function of Men1 gene or its encoded proteins for the purpose of treating diabetes and screening and researching agents capable of inhibiting expression or function of Men1 gene or its encoded proteins for use in the treatment and diagnosis of diabetes.

Abstract: Provided are a single-stranded nucleic acid aptamer simultaneously and specifically binding to various types of microorganisms, and a method of manufacturing the nucleic acid aptamer. For example, provided are a probe that is capable of simultaneously detecting or diagnosing a variety of microorganisms, and a method of manufacturing an aptamer having characteristics of such a probe.

Abstract: Described herein are compositions comprising polymers prepared by melt polycondensation of 2-hydroxyalkyl acids. Methods of making and using the compositions are also disclosed.

Abstract: Disclosed are methods and compositions for siRNA-mediated therapy of mammalian diseases. In particular, compositions and methods are disclosed for treatment of therapy-resistant human breast cancers. In exemplary embodiments, siRNA molecules are presented that effectively knock down gene expression of one or more polynucleotides in breast cancer tumor initiating cells.

Abstract: A novel network of tumorigenic prognostic factors is identified that plays a critical role in advanced pancreatic cancer (PC) pathogenesis. This interactome is interconnected through a central tumor suppressive microRNA, miR-198, which is able to both directly and indirectly modulate expression of the various members of this network to alter the molecular makeup of pancreatic tumors, with important clinical implications. When this tumor signature network is intact, miR-198 expression is reduced and patient survival is dismal; patients with higher miR-198 present an altered tumor signature network, better prognosis and increased survival. Further, according to the present disclosure, MiR-198 replacement reverses tumorigenicity in vitro and in vivo.

Abstract: Polymers produced by ring opening polymerization which comprises an amino group that can be used in compositions to deliver a nucleic acid such as a miRNA or a siRNA. In some embodiments, compositions which comprise the polymers described herein and a nucleic acid are also provided herein. In some embodiments, these compositions are used to silence one or more genes in vivo or treat a disease or disorder.

Abstract: The present invention relates to an active substance release system containing two compounds. The first compound comprises a nanoparticle, combined with an oligonucleotide inhibition strand that is hybridized with a catalytically active nucleic acid. The second compound comprises a carrier, combined with a substrate molecule that is coupled to a therapeutic active substance. By means of external stimulation, the catalytically active nucleic acid of the first compound is released and specifically binds to the substrate molecule of the second compound. This leads to cleavage of the substrate molecule, whereby the active substance bound thereto is released.

Abstract: An aptamer-based SERS detection technique that directly monitors an aptamer-analyte capture event by generating spectroscopic information regarding the identity of the analyte that has been bound to the aptamer from a complex biological sample. A reproducible SERS spectrum is measured for an aptamer-analyte complex formed on a metal surface and this spectral information is used directly to identify the specific aptamer-analyte complex and optionally also to quantify the analyte in the sample, thus enabling discrimination between true and false positives in quantitative analyte assays on complex biological samples. In one embodiment the aptamer is attached directly to the metal surface and surrounded by a self-assembled monolayer (SAM) of amphiphilic molecules. In an alternative embodiment the metal surface is coated with a SAM and the aptamer is attached to the amphiphilic molecules of the SAM.

Abstract: This invention provides a Chinese medicinal formula comprising Atractylodis Macrocephalae Rhizoma, Glycyrrhizae Radix et Rhizoma, Angelicae sinensis Radix, Paeoniae Radix Alba and Poria for skin care. It further relates to a method for preparing bioactive fraction of this formula and to the applications of the bioactive fraction in skin-whitening and anti-skin-aging.

Abstract: The present invention provides a pharmaceutical agent which causes skipping of the 55th, 45th, 50th or 44th exon in the human dystrophin gene with a high efficiency. The present invention provides an oligomer which efficiently enables to cause skipping of the 55th, 45th, 50th or 44th exon in the human dystrophin gene.