Abstract: The present invention relates to the design and development of recombinant protein for the delivery of silencer RNA complex to mediate RNA interference since it represents a novel therapeutic approach to modulate several neurodegenerative disease-related genes across the blood-brain barrier (BBB). To overcome challenges due to this barrier for biologics and other biological complex, the present invention describes a method wherein peptide having sequence GGGGHLNILSTLWKYRC represented by SEQ ID NO. 9 known to target specific gangliosides was linked to a double-stranded RNA binding protein to bind and deliver silencer RNA to the brain parenchyma. The designed fusion protein comprising a double-stranded RNA-binding domain (dsRBD) of human Trans Activation response element (TAR) RNA Binding Protein (TARBP2) and a brain targeting peptide sequence that binds GM1. Conformation-specific binding of TARBP2 domain to silencer RNA results in the formation of homogenous serum-stable complex with GM1 targeting potential.

Abstract: A conjugate for delivery of drugs, such as genetic drugs, [e.g., siRNA, dsiRNA, or antisense oligonucleotides (ASO)] across biological membranes is provided. The conjugates of the Invention are capable of delivering drugs in both presence and absence of plasma proteins.

Abstract: Disclosed are double-stranded antisense nucleic acid complexes that can efficiently alter the processing of RNA in a cell via an antisense effect, and methods for using the same. One method comprises contacting with the cell a double-stranded nucleic acid complex comprising: a first nucleic acid strand annealed to a second nucleic acid strand, wherein: the first nucleic acid strand comprises (i) nucleotides independently selected from natural DNA nucleotides, modified DNA nucleotides, and nucleotide analogs, (ii) no regions that have 4 or more consecutive natural DNA nucleotides, (iii) the total number of natural DNA nucleotides, modified DNA nucleotides, and nucleotide analogs in the first nucleic acid strand is from 8 to 100, and (iv) the first nucleic acid strand is capable of hybridizing to RNA inside of the cell; and the second nucleic acid strand comprises nucleotides independently selected from natural RNA nucleotides, modified RNA nucleotides, and nucleotide analogs.

Abstract: Multiplex immunoassays utilize the differential affinities among the conjugation pairs between the capture ligands and target analytes are proposed. Window magnetic-assisted rapid aptamer selection (window-MARAS) methods for selecting aptamers with desirable affinity toward the target analytes and methods for generating reagents for multiplex immunoassays or multiplex detection in one assay by utilizing the selected aptamers as capture ligands in reagents are described and used to demonstrate the feasibility of multiplex immunoassays based on the differential affinity of conjugation pairs between the capture ligands and target analytes.

Abstract: Molecules of general Formula (I): able to bind to native polyribosomes engaged in active protein synthesis. The disclosure relates also to the use of the molecules of general Formula (I) for isolating at least one active ribosome from a biological sample, and for ribosome profiling, as well as kits for isolating at least one active ribosome from a biological sample.

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: The disclosure provides for dendronized polymers, and the use of the polymers as carriers for the intracellular delivery of nucleic acids.

Abstract: The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing and/or delaying DMD or BMD.

Abstract: The invention relates to methods for diagnosing multiple sclerosis with miRNA markers. Diagnosis of multiple sclerosis (MS) can be challenging in patients with atypical presentations and during a first neurological deficit possibly related to inflammatory demyelination. Towards the identification of biomarkers for diagnosis of MS, a comprehensive analysis of miRNA expression patterns was obtained. Significantly deregulated miRNAs were identified, which have previously not been related to MS according to the microRNA disease database. These miRNAs could potentially serve as future diagnostic biomarkers for MS and help in diagnosis, monitoring disease activity, and evaluation of treatment responses in patients with MS.

Abstract: A method of reducing a subject's plasma triglyceride level, comprising administering to a subject in need thereof a gamma-secretase inhibitor in an amount effective to reduce the subject's plasma triglyceride level.

Abstract: Several miRNAs are described that are useful in diagnosing and treating prostate cancer, or pancreatic cancer. The miRNAs bind with targets that are associated with prostate cancer or pancreatic cancer. This permits the identification of those targets as well as a treatment methodology for prostate cancer.

Abstract: Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal. Such methods are useful to treat, prevent, ameliorate, or slow progression of neurodegenerative diseases in an individual in need thereof.

Abstract: New cationic lipids are provided that are useful for delivering macromolecules, such as nucleic acids, into eukaryotic cells. The lipids can be used alone, in combination with other lipids and/or in combination with other transfection enhancing reagents to prepare transfection complexes.

Abstract: Methods and kits for GPP-targeting, e.g., for the treatment of oncogenic Kras-associated cancers, and methods for determining the efficacy of those methods are provided.

Abstract: Blunting the activity of the P2Y2 receptor results in a resistance to diet-induced obesity, an increased metabolic rate, and a better glucose tolerance. Compounds that inhibit the puringeric P2Y2 receptor are useful for treating disorders associated with diabetes, treating obesity, and increasing metabolism (e.g., fatty acid metabolism).

Abstract: Materials and methods are provided for producing and using aptamers useful as oncology therapeutics capable of binding to PDGF, PDGF isoforms, PDGF receptor, VEGF, and VEGF receptor or any combination thereof with great affinity and specificity. The compositions of the present invention are particularly useful in solid tumor therapy and can be used alone or in combination with known cytotoxic agents for the treatment of solid tumors. Also disclosed are aptamers having one or more CpG motifs embedded therein or appended thereto.

Abstract: The present invention is related to a nucleic acid molecule capable of binding to SDF-1, preferably capable of inhibiting SDF-1, whereby the nucleic acid molecule is for use in a method for the treatment and/or prevention of a disease or disorder, for use in a method for the treatment of a subject suffering from a disease or disorder or being at risk of developing a disease or disorder as an adjunct therapy, or for use as a medicament for the treatment and/or prevention of a disease or disorder, whereby the disease or disorder is cancer.

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LECT2 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of LECT2.

Abstract: A process and molecular biomarker for platelets stored in blood banks provides for efficient verification of which platelet concentrate bags are in good condition for transfusion by measuring, by real-time polymerase chain reaction, expression levels of hsa-miR-127 and hsa-miR-320a microRNAs as the first process and biomarker predictor of platelet's cell aging during storage and the consequent presence of storage lesions as a quality test of these bags.

Abstract: The present invention relates to a therapeutic compound comprising: an agent that inhibits the activity of at least one gene selected from the group consisting of MAF, MEOX2, SIX2 and homologs thereof having at least 50% identity with said genes and/or an agent that enhances the activity of at least one gene selected from the group consisting of CREB5, E2F1, EGR2, HIC1, IRF7, JUN, MYC, SRF, STAT4, TCF4, FOXS1, GLI1, SOX9 and homologs thereof having at least 50% identity with said gene for use in the treatment of wounds, preferably chronic wounds.