Abstract: Methods and uses for diagnosing and treating anxiety disorders are encompassed, wherein diagnosis and treatment may be based upon an assessment of genetic alterations in metabotropic glutamate receptor (mGluR) network genes and wherein treatment is with nonspecific activators of mGluRs such as fasoracetam.

Abstract: The present application relates to plasma cells and plasma cell precursors that express a macromolecule, such as a protein, protein mimetic or a peptide and compositions comprising these plasma cells or plasma cell precursors. The application further relates to methods of using and making the plasma cells and plasma cell precursors that express the macromolecule. Methods of treatment comprising administering the plasma cells or plasma cell precursors are also contemplated.

Abstract: The present invention relates to a tissue-specific promoter system for expressing microRNA (miRNA) for RNA interference-based methods of gene therapy. In these systems, the miRNA will inhibit gene expression or replace natural miRNA expression using microRNA.

Abstract: Polynucleotides and vectors can be used for the expression of a transgene in cells, such as liver cells. The expression of the transgene from the polynucleotides and vectors can be useful in gene therapy. Various methods can be used for expressing the transgene from the polynucleotides and vectors in liver cells.

Abstract: Early detection of lysosomal storage diseases (LSDs) including Mucopolysaccharidosis Type I (MPS I) and Pompe Disease can greatly improve patient outcome as each disease can be fatal once symptoms emerge. Screening for MPS I and Pompe Disease using biological samples including dried blood spots (DBS), buccal swab, peripheral blood mononuclear cells (PBMCs), or white blood cells (WBCs) is described. The disclosed methods and assays provide a robust way to screen newborns for LSDs. The disclosed methods and assays can also allow rapid prediction of whether a patient with LSD will develop an immune response to enzyme replacement therapy (ERT), thus improving treatment for patients with LSDs. The disclosed methods and assays can also further reduce the number of false positives caused by pseudo deficiency cases of LSD, such as MPS I and Pompe Disease.

Abstract: An anti-ANGPTL3 antibody or an antigen-binding fragment thereof. The present invention provides an anti-ANGPTL3 antibody or an antigen-binding fragment thereof, wherein a heavy chain variable region includes complementarity determining regions (CDRs), including CDR-H1 having an amino acid sequence as set forth in SEQ ID No. 1, CDR-H2 having an amino acid sequence as set forth in SEQ ID No. 2, and CDR-H3 having an amino acid sequence as set forth in SEQ ID No. 3; and the CDRs of a light chain variable region includes CDR-L1 having an amino acid sequence as set forth in SEQ ID No. 4, CDR-L2 having an amino acid sequence as set forth in SEQ ID No. 5, and CDR-L3 having an amino acid sequence as set forth in SEQ ID No. 6. The antibody or the antigen-binding fragment thereof provided in the present invention can specifically recognize ANGPTL3.

Abstract: Disclosed herein are products, methods, and uses for treating, ameliorating, delaying the progression of, and/or preventing a muscular dystrophy or a cancer including, but not limited to, facioscapulohumeral muscular dystrophy (FSHD) or a cancer associated with DUX4 expression or overexpression. More particularly, disclosed herein are RNA interference-based products, methods, and uses for inhibiting or downregulating the expression of double homeobox 4 (DUX4). Even more particularly, the disclosure provides microRNA (miRNA) for inhibiting or downregulating the expression of DUX4 and methods of using said miRNA to inhibit or downregulate DUX4 expression in cells and/or in cells of a subject having a muscular dystrophy or a cancer including, but not limited to, FSHD or a cancer associated with DUX4 expression or overexpression.

Abstract: Aspects of the invention described herein relate to methods of making and using inducible promoters for transgene expression. The inducible promoters are derived from the NFAT-RE inducible system and are used to improve or enhance T cell survival and proliferation.

Abstract: Some embodiments of the invention include a nucleic acid molecule comprising natural nucleotides, non-natural nucleotides, an LNA which comprises one or more RNA core molecules, or an RNA molecule which comprises more than one RNA core molecule. Some embodiments of the invention include a nucleic acid molecule comprising an RNA molecule which comprises more than one RNA core molecule. Other embodiments of the invention include a nucleic acid molecule comprising a DNA molecule encoding the RNA molecule (e.g., vector or viral vector). Other embodiments include compositions or pharmaceutical compositions that comprise the nucleic acid molecule. Some embodiments of the invention comprise reducing miR-143 in a cell. Other embodiments of the invention include methods to deliver a protein across the BBB. Other embodiments include methods for treating disease (e.g., LSD), neuronopathic disease, neurodegenerative disease, Hurler syndrome, or MPS I). Additional embodiments of the invention are also discussed herein.

Abstract: This invention relates to methods of treating and ameliorating congenital and neonatal hyperinsulinism and post-prandial hypoglycemia, comprising the step of administering an antagonist of the Glucagon-Like Peptide-1 (GLP-1) receptor, e.g. a GLP-1 fragment or analogue thereof.

Abstract: The present disclosure is directed to antibodies binding to Glypican 2 and methods of using such antibodies to treat cancers that express or overexpress the Glypican 2 antigen.

Abstract: Compositions and methods for the diagnosis and treatment of Covid 19 are disclosed.

Abstract: The present disclosure relates to recombinant adeno-associated vims (rAAV) delivery of a neurotrophin 3 (NT-3) polynucleotide. The disclosure provides rAAV and methods of using the rAAV for NT-3 gene therapy to improve muscle strength, stimulate muscle growth and to treat muscle wasting disorders, such as muscular dystrophy and Charcot-Marie-Tooth neuropathy.

Abstract: This disclosure provides isolated or recombinant polypeptides that are useful to vaccinate individuals suffering from chronic/recurrent biofilm disease or as a therapeutic for those with an existing infection. The individual's immune system will then naturally generate antibodies which prevent or clear these bacteria from the host by interfering with the construction and or maintenance of a functional protective biofilm. Alternatively, antibodies to the polypeptides can be administered to treat or prevent infection. Bacteria that are released from the biofilm by our technology are more readily cleared by the remainder of the host's immune system.

Abstract: Methods for diagnosing and treating conduct disorder are encompassed, wherein diagnosis and treatment may be based upon an assessment of genetic alterations in metabotropic glutamate receptor (mGluR) network genes and wherein treatment is with nonspecific activators of mGluRs such as fasoracetam.

Abstract: In Hybrid and truncated immune cell proteins are described. Hybrid proteins are stimulatory and include an extracellular domain of one stimulatory immune cell protein, an intracellular domain of a different stimulatory immune cell protein, and a transmembrane domain linking the extracellular domain to the intracellular domain. Truncated proteins include an immune cell receptor ligand and a transmembrane domain but lack a functional intracellular domain. The hybrid and truncated proteins can be used to modulate and/or diversify immune cell activation in the fight against cancers and infectious diseases, among other uses.

Abstract: A frequency encoded source imaging system includes an EEG or MEG sensor array and a processing system for analyzing the signals from the sensor array in at least two different frequency bands, where the analysis is localized with respect to a three-dimensional grid corresponding to the portion of the human body. Alternately, a frequency encoded source imaging system includes an EEG or MEG sensor array and a processing system for analyzing the signals from the sensor array in a high-definition frequency band comprising frequencies greater than 70 Hz, where the analysis is localized with respect to a three-dimensional grid corresponding to the portion of the human body.

Abstract: Provided herein are methods and kits for collecting and/or detecting biological materials from the skin of subjects. Methods and kits for determining a biological profile of a target skin site of subjects are also provided herein.

Abstract: A simulation method for a chronic atrophic gastritis (CAG) lesion includes: (1) taking a metaplasia lesion stage as a simulation object, (2) selecting a simulation form of spasmolytic polypeptide-expressing metaplasia (SPEM), and (3) conditionally deleting gene associated with retinoid-IFN-induced mortality-19 (GRIM-19) from gastric mucosal parietal cells. The present disclosure successfully simulates the SPEM, an initial metaplasia response after a gastric mucosal injury and the initial metaplasia response can progress into intestinal metaplasia (IM) and even gastric cancer (GC) under the continuous stimulation of chronic inflammation.

Abstract: The disclosure provides for methods of predicting disease progression comprising detecting a modification of one or more disease markers in a glial cell or neuronal cell generated from a skin cell of the subject. The disclosed methods include methods of identifying subjects that are responsive to a therapeutic agent and methods of determining effectiveness of a therapeutic agent.