Abstract: Described are methods for early noninvasive or minimally invasive detection of pathological changes in organ systems/organs/tissues/cells by quantifying organ system-/organ-/tissue-/cells type-enriched miRNA in bodily fluids.

Abstract: The present invention relates to a method for quantitating an analyte having a wide range concentration in a single assay without having to dilute the sample and repeating the assay. The key feature of the invention is having two cycles of events including sample binding to probe, binding reactions, and detection. After the first cycle of binding and detecting, the probe is dipped into the same sample vessel to bind additional analyte in the sample vessel at a condition that is more favorable to binding than the condition in the first cycle.

Abstract: The present invention provides methods, devices and kits for detecting binding between two molecules using a liquid crystal.

Abstract: This invention covers a new molecular recognition system, where duplexes of DNA-like molecules comprise segments built from nucleotides that carry only a small pyrimidine-like analog, and where the segments pair by pyrimidine analog:pyrimidine analog “skinny” pairing. This pairing retains hydrogen bonding complementarity. Further, this invention relates to processes for preparing those duplexes, and processes that use such duplexes as primer:template complexes for reactions catalyzed by DNA polymerases.

Abstract: Based at least in part on an understanding of the mechanisms by which small RNAs (e.g., naturally-occurring miRNAs) mediate RNA silencing in plants, rules have been established for determining, for example, the degree of complementarity required between an RNAi-mediating agent and its target, i.e., whether mismatches are tolerated, the number of mismatches tolerated, the effect of the position of the mismatches, etc. Such rules are useful, in particular, in the design of improved RNAi-mediating agents which allow for more exact control of the efficacy of RNA silencing.

Abstract: The present disclosure provides compounds comprising oligonucleotides complementary to a portion of the IKBKAP gene. Certain such compounds are useful for hybridizing to a portion of the IKBKAP gene, including but not limited to a portion of the IKBKAP gene in a cell. In certain embodiments, such hybridization results in modulation of splicing of the IKBKAP gene. In certain embodiments, the IKBKAP gene includes a mutation that results in defective splicing and a truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in a decrease in the amount of defective splicing and truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in an increase in the amount of normal splicing and functional, full-length IKAP protein. In certain embodiments, oligonucleotides are used to treat Familial Dysautonomia.

Abstract: The present invention encompasses a class of compounds known as splice modulating oligonucleotides (SMOs) that modulate pre-mRNA splicing, thereby affecting expression and functionality of a specific protein in a cell. The present invention further provides compositions and methods for modulating pre-mRNA splicing using a SMO of the invention to abrogate disease-causing mutations in a protein. Accordingly, the present invention provides compositions and methods of treating a subject at risk of, susceptible to, or having a disease, disorder, or condition associated with aberrant or unwanted target pre-mRNA expression or activity.

Abstract: Disclosed is a lateral flow capillary device and uses thereof comprising a unipath bibulous capillary flow matrix and at least two reservoirs each in fluid communication with the capillary flow matrix wherein a reservoir contacts the capillary flow matrix through a passage having a rim pressing the matrix. The pressure that the rim applies on the matrix prevents leakage of liquids out of the capillary flow matrix at the reservoir/matrix interface, allowing accurate sequential draining of liquid from the reservoirs.

Abstract: A method for identifying a cluster or sub-cluster of microRNAs (miRNAs) that provides a signature profile for differentiating cells grown in one type of culture model from cells grown in another type of culture model. Also, kits and methods for evaluating, selecting, and/or characterizing tissue culture models using the miRNA profiles and a cluster or sub-cluster of miRNAs that provides the signature profile. Also, a method for identifying a putative mRNA target of a miRNA for evaluating and targeting with a drug candidate by using the cluster or sub-cluster of miRNAs and a method for selecting a culture model for use as a drug platform for screening a candidate molecule for activity against tumor cells in tumor stroma.

Abstract: The present application relates to a method for the targeted formation of heterochromatin and/or induction of epigenetic gene silencing in a cell using a small RNA, said method comprising the step of inhibiting the Paf1 complex in said cell and the step of contacting said cell with a small RNA targeted to a region of the genome of the cell, said region being the region where heterochromatin formation and/or induction of epigenetic gene silencing should be induced.

Abstract: Described is a lipid nanoparticle composition that includes a macromolecule conjugated to a polymer and a targeting agent. The composition can include a therapeutic agent. The therapeutic agent can be an antisense oligonucleotide (ASO). Exemplary ASOs are targeted to a portion of a nucleic acid encoding Akt-1, and which modulates the expression of Akt-1; or targeted to a portion of a nucleic acid encoding HIF-1, and which modulates the expression of HIF-1. Also described is a lipid nanoparticle composition that includes a macromolecule conjugated to a polymer and a therapeutic agent that is an ASO such as an ASO targeted to a portion of a nucleic acid encoding Akt-1, and which modulates the expression of Akt-1 or an ASO targeted to a portion of a nucleic acid encoding HIF-1, and which modulates the expression of HIF-1. Pharmaceutical formulations, methods of making the lipid nanoparticles, and methods of using the lipid nanoparticles, for example for treating cancers, are also disclosed.

Abstract: Disclosed are devices for collecting and stabilizing blood that contain a blood stabilization agent which includes variegin or an analog thereof, a polysulfated disaccharide, or a combination thereof, each in an amount effective to stabilize blood. Methods of making and using the devices, and kits containing the devices, are also provided.

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: An aptamer specific to ovarian cancer and a detection method for ovarian cancer are provided. The aptamer includes a following nucleotide sequence: 5?-ncaaannncnnnnanncnnnnnnnnnnngaannnannngg-3?, wherein n is a nucleotide independently selected from “a,” “t,” “c,” and “g.

Abstract: Embodiments provide methods and compositions related to determining treatments for colorectal cancer patients by detection and analysis of the expression level of miRNA such as miR-320e in the patients. Embodiments provide predictive, prognostic and/or diagnostics methods by identifying miRNAs that are useful for clinical management of cancer patients, particularly colorectal cancer patients or patients at risk or determined to have colorectal cancer. Methods and compositions are based, in part, on the discovery that expression of certain miRNAs in cancer patients is associated with advancing cancer stages and/or can predict the responsiveness of cancer therapy, and can, therefore, provide basis for designing treatment strategies. In particular embodiments, the miRNA molecule is miR-320, particularly miR-320e.

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: Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for TTR.

Abstract: Provided is an RNA interference-inducing nucleic acid comprising at least one nucleic acid strand, the at least one nucleic acid strand comprising a modification substituted to a spacer, which is unable to form a base pair, in the 5? end or the 3? end region. The RNA interference-inducing nucleic acid is a modified form of nucleotide provided to prevent off-target effects, offering a method to selectively repress target gene expression. The RNA interference-inducing nucleic acid provides modified forms with target selectivity and specificity as a method to block the off-target effects while silencing the target gene expression, whereas the usage of conventional RNA interference-inducing nucleic acids cause inaccuracy and adverse effects through off-targets, thereby the RNA interference-inducing nucleic acid was offered to solve the problem, wherein it will be widely used as a method for repressing gene expression in research and for gene therapy without concerning the off-target effects.

Abstract: Disclosed herein is a modular composition comprising 1) an oligonucleotide; 2) one or more tetraGalNAc ligands of Formula (I), which may be the same or different; optionally, 3) one or more linkers, which may be the same or different; 4) one or more peptides independently selected from Table 3, which may be the same or different; and optionally, 5) one or more targeting ligands, solubilizing agents, pharmacokinetics enhancing agents, lipids, and/or masking agents.

Abstract: Disclosed are compositions and methods that use lysine demethylase inhibitors for inhibiting the growth of cancer stem cells or tumor initiating cells, for enhancing the biological effects of chemotherapeutic drugs or irradiation on cancer cells and/or for preventing cancer recurrence.