Abstract: The disclosed embodiments concern methods, apparatus, systems and computer program products for determining sequences of interest using unique molecular index (UMI) sequences that are uniquely associable with individual polynucleotide fragments, including sequences with low allele frequencies and long sequence length. In some implementations, the UMIs include both physical UMIs and virtual UMIs. In some implementations, the unique molecular index sequences include non-random sequences. System, apparatus, and computer program products are also provided for determining a sequence of interest implementing the methods disclosed.

Abstract: The microRNA dyslipidemia inhibitor includes methods of administering a miRNA capable of inhibiting dyslipidemia to a subject in need thereof. The methods of the miRNA dyslipidemia inhibitor may include administration of miRNA-103 to a subject in need thereof. The administration of miRNA-103 may inhibit ANGPTL8 activity, thereby reducing ANGPTL8's inhibition of lipoprotein lipase activity and increasing triglyceride degradation. The administration of miRNA-103 may be used to treat or prevent lipid metabolism disorders including dyslipidemia and dyslipidemia associated diseases, such as hyperlipidemia, atherosclerosis, heart disease, and the like.

Abstract: Methods of promoting hypoxia or the hypoxia response for the treatment or prevention of mitochondrial dysfunction and oxidative stress disorders are described. Methods for screening for targets of mitochondrial dysfunction and oxidative stress disorders are also described.

Abstract: Provided are compositions and methods comprising two-tailed siRNAs (tt-siRNAs) that exhibit unprecedented cellular uptake and silencing. Also provided are methods of treating neurological and other diseases with the two-tailed siRNAs of the invention.

Abstract: The present invention generally relates to systems and methods to create stable emulsions with low rates of exchange of molecules between microdroplets.

Abstract: An assay device including a loading zone for a sample that may contain an analyte, an activator in communication with the loading zone from which at least one activator is displaced by presence of the analyte, an amplifier in an inactive state in communication with the activator that becomes activated in the presence of the activator, a biomatrix barrier in communication with the activated amplifier that is degraded or modified by the activated amplifier, and an indicator responsive to the degradation or modification of the biomatrix barrier, which in turn reflects a concentration of the analyte in the sample loaded on the device. The selection of analyte or displacement of the activator by presence of the analyte may occur off-platform and/or may rely on use of non-covalent interactions. The activator may include an enzyme or other reagent that activates the amplifier. Also, associated methods.

Abstract: A method of treating hyperglycemia, diabetes, metabolic syndrome, insulin resistance (insulin insensitivity), impaired glucose tolerance, high glucose levels, pulmonary hypertension, and/or a condition arising from any of the foregoing in a patient is provided. The method comprises knocking down mARC2 or mARC1 expression in the patient, or otherwise decreasing mARC2 and mARC1 activity in the patient.

Abstract: Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2 that comprises exon 7. Methods for modulating expression of SMN1 or SMN2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of SMN1 or SMN2.

Abstract: The present invention relates to a method for detecting cell death using a luminescent compound; to the luminescent compounds for particular uses; to a kit comprising said compounds and to a protein. The method is applicable for detecting cell death, essentially regardless of the mechanism through which cell death occurred or is occurring and is therefore not limited e.g. to detecting cell death resulting from only one mechanism selected from apoptosis and necrosis.

Abstract: Provided are a method for preventing or treating obesity by using a composition including an inhibitor of expression of the Hoga1 gene or an inhibitor of activity of the Hoga1 protein as an active ingredient, and a method of screening a preventive or therapeutic agent for obesity by using the composition.

Abstract: Uses of ethyl 5,11-dihydroindolo[3,2-b]carbazole-6-carboxylate as a fluorescent ligand probe, preferably in a binding assay for quantitative analysis in combination with a recombinant aryl hydrocarbon receptor (AhR) protein. A method for detection or quantitative analysis of suspected aryl hydrocarbon receptor (AhR) ligands in a sample, the method comprising the steps of: (a) providing a sample possibly containing at least one known or unknown AhR ligand; (b) mixing said sample with a composition comprising a recombinant AhR protein bound to ethyl 5,11-dihydroindolo[3,2-b]carbazole-6-carboxylate; and (c) determining the presence or the total amount of said at least one known or unknown AhR ligand in the sample by fluorescence spectroscopy. Use e.g.

Abstract: Provided herein are methods and assays for isolating and culturing seborrheic keratosis cells ex vivo. Also provided herein are screening assays using cultured seborrheic keratosis cells and methods for treating seborrheic keratosis in a subject.

Abstract: Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.

Abstract: The present invention relates to the use of a DBait molecules by systemic routes without any combination with an endosomolytic agent.

Abstract: The present invention relates to a method for measuring the cholesterol uptake capacity of lipoproteins. The present invention also relates to a reagent kit for measuring the cholesterol uptake capacity of lipoproteins. The present invention further relates to a tagged cholesterol which can be used in the method and the reagent kit.

Abstract: This application relates to potent oligonucleotides useful for reducing HBsAg expression and treating HBV infections.

Abstract: Ribozymes exhibiting tRNA synthetase activity and substrate specificity, as well as methods for engineering and producing the same, are disclosed. The ribozymes of the present disclosure comprise a T-box module fused with a flexizyme module. The flexizyme module provides high promiscuity with respect to amino acid substrates and the T-box module provides tRNA substrate specificity, which may be engineered as desired. Systems are also described for aminoacylation of suppressor tRNAs with unnatural amino acids (uAAs), such systems comprising the ribozyme previously mentioned, suppressor tRNA, and the desired uAA(s). Methods for incorporating a uAA into a growing polypeptide chain using the ribozyme hereof are also provided.

Abstract: Methods for identifying a compound that alters fluorescence resonance energy transfer (FRET) of a protein. The methods include use of a genetically engineered cell that includes a target protein. The target protein includes one or more heterologous domains. In one embodiment, a target protein includes two heterologous domains, and in another embodiment, the target protein includes a heterologous domain and the cell further includes a second protein that includes a heterologous domain. A heterologous domain may include a chromophore or an amino acid to which a fluorescent dye attaches. The fluorescence lifetime of one or more chromophore, one or more fluorescent dye, or the combination thereof, is measured after contacting the cell with a compound A difference between the fluorescence lifetime in the presence of the test compound and the fluorescence lifetime in the absence of the test compound indicates that the test compound alters the FRET of the target protein.

Abstract: Pharmaceutical compositions are provided which include a microRNA pool capable of promoting, stimulating or increasing neuronal differentiation, as well as methods for treating, ameliorating and/or preventing depression or diseases where damage to nervous tissue occurs, such as neurological diseases, by means of such microRNA pool.

Abstract: A novel method for preparing sequence-verified oligonucleotides is disclosed. In particular, the invention relates to a simple, affordable, and scalable method that combines high-throughput mating of yeast clones, a unique selectable system for combining DNA sequences in yeast, and next-generation sequencing. This method allows sequence-verified oligonucleotides to be readily isolated from complex libraries.