Abstract: The invention relates to a gene transfer-based method to protect a subject from Sjogren's syndrome. The method comprises administering to the subject an AAV virion comprising an AAV vector that encodes aquaporin-1 (AQP-1) protein. Also provided are AQP-1 proteins and nucleic acid molecules that encode such proteins. Also provided are AAV vectors and AAV virions that encode an AQP-1 protein.

Abstract: Substituted phenylpyrrolecarboxamide compounds such as those represented by Formula A can be used in the treatment of HIV infection and related conditions.

Abstract: The present invention is directed to methods of inhibiting p97 and compounds and compositions useful in such methods. Diseases and conditions the can be treated with the compounds and compositions of the invention include, but are not limited to, cancer and neurodegenerative disorders susceptible to treatment by inhibition of p97.

Abstract: Provided herein are capillaries for use in an electrophoretic separations. The capillaries can comprise an elongated tubular wall defining a path for fluid flow from an inlet to a tapered outlet; and a decoupler junction positioned within the elongated tubular wall upstream of the tapered outlet. The decoupler junction can comprise a plurality of microchannels penetrating through the elongated tubular wall and an ion-permeable polymer coating the plurality of microchannels. Also provided are sheathless interfaces for coupling capillary electrophoresis (CE) with mass spectrometry that employ these capillaries.

Abstract: Regional, tumor-targeted, cytotoxic therapy, such as D2C7-immunotoxin (D2C7-IT), not only specifically target and destroy tumor cells, but in the process initiate immune events that promote an in situ vaccine effect. The antitumor effects are amplified by immune checkpoint blockade which engenders a long-term systemic immune response that effectively eliminates all tumor cells.

Abstract: Provided are methods for treating GLUT1 and related brain energy deficiencies comprising administering odd-carbon fatty acid sources, e.g., C5 or C7 fatty acid sources, and related compositions.

Abstract: A therapeutic apparatus (900, 1000) comprising a high intensity focused ultrasound system (904) for heating a target zone (940, 1022). The therapeutic apparatus further comprises a magnetic resonance imaging system (902). The therapeutic apparatus further comprises a memory (952) containing machine executable instructions (980, 982, 984, 986, 988, 990) for execution by a processor (944). Execution of the instructions cause the processor to: generate (702, 802) heating commands (964) which cause the high intensity focused ultrasound system to sonicate the subject; repeatedly acquire (704, 804) magnetic resonance data (954) during execution of the heating commands; repeatedly calculate (706, 806) a spatially dependent parameter (970); and repeatedly modify (708, 808) the heating commands in accordance with the spatially dependent parameter such that within the target zone the spatially dependent parameter remains below a first predetermined threshold and above a second predetermined threshold.

Abstract: The present disclosure relates to certain amine derivatives of fused bicyclic heterocycles that inhibit the amine reuptake function of the biogenic amine transporters, dopamine transporter (DAT), serotonin transporter (SERT) and norepinephrine transporter (NET). Compounds of the present disclosure are potent inhibitors of the reuptake of dopamine (DA), serotonin (5-hydroxytryptamine, 5-HT) and norepinephrine (NE) with full or partial maximal efficacy. The compounds with partial maximal efficacy in inhibiting reuptake of all three biogenic amines are herein referred to as partial triple uptake inhibitors (PTRIs). Compounds of the present disclosure are useful for treating depression, pain and substance abuse and relapse to substance abuse and addiction to substances such as cocaine, methamphetamine, nicotine and alcohol. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

Abstract: Methods are disclosed for treating a subject with a vascular or cardiac disorder associated with oxidative stress. Methods are disclosed for treating a subject with GNE myopathy that has impaired cardiac function. These methods include administering to the subject a therapeutically effective amount of a sialic acid precursor, sialic acid, or one or more sialylated compounds, mannosamine, N-acetyl mannosamine or a derivative thereof. In other embodiments, methods are disclosed for detecting a disorder associated with oxidative stress.

Abstract: We have constructed a polynucleotide encoding a bispecific antibody engaging molecule which has one arm that specifically engages a tumor cell which expresses the human EGFRvIII mutant protein on its surface, and a second arm that specifically engages T cell activation ligand CD3. The polynucleotide is codon optimized for expression in CHO cells. The subunits of the engaging molecules are organized to achieve greater efficiency. These are therapeutic agents.

Abstract: A composition which is reversible inhibitor of at least one neuron-specific PDZ domain comprising wherein R is a molecular transporter with or without a linker amino acid; R1 is at least about one amino acid covalently bound; and, R2 is isoleucine, leucine, alanine, phenylalanine, or valine, and methods of use.

Abstract: A therapeutic composition for treating brain injury comprising a polyarginine peptide of from 5 to 9 arginines (SEQ ID NO: 1), and further comprising 1 or more terminal cysteines. The composition is administered in therapeutically effective dosages prophylactically or as soon as possible post-injury in treating neuronal injury.

Abstract: The present invention provides small molecule inhibitors of BMP signaling. These compounds may be used to modulate cell growth, differentiation, proliferation, and apoptosis, and thus may be useful for treating diseases or conditions associated with BMP signaling, including inflammation, cardiovascular disease, hematological disease, cancer, and bone disorders, as well as for modulating cellular differentiation and/or proliferation. These compounds may also be used to reduce circulating levels of ApoB-100 or LDL and treat or prevent acquired or congenital hypercholesterolemia or hyperlipoproteinemia; diseases, disorders, or syndromes associated with defects in lipid absorption or metabolism; or diseases, disorders, or syndromes caused by hyperlipidemia.

Abstract: The present invention relates, in general, to HIV-1 and, in particular, to broadly neutralizing HIV-1 antibodies, and to HIV-1 immunogens and to methods of using such immunogens to induce the production of broadly neutralizing HIV-1 antibodies in a subject (e.g., a human).

Abstract: A method for generating a perfusion weighted image using ASL with segmented acquisitions includes dividing an anatomical area of interest into slices and performing an EPI acquisition process using an MRI system to acquire a control image dataset representative of the slices. An ASL preparation process is performed using the MRI system to magnetically label protons in arterial blood water upstream from the anatomical area of interest. Following a first time period, a multi-band EPI acquisition process is performed using the MRI system to acquire a first labeled image dataset representative of a first subset of the slices. Following a second time period, another multi-band EPI acquisition process is performed using the MRI system to acquire a second labeled image dataset representative of a second subset of the slices. A perfusion weighted image is generated by subtracting the first and second labeled image dataset from the control image dataset.

Abstract: The present invention generally relates to a method for determining the dynamic viscoelastic properties of cells, more particularly to a method for rapidly determining the dynamic viscoelastic properties of healthy and unhealthy cells by determining the phase shift be the application of a modulating force to the cells and the cells' response to the modulating force.

Abstract: The disclosure provides pharmaceutical preparations containing (2R,6R)-hydroxynorketamine, or (R)- or (S)-dehydronorketamine, or other stereoisomeric dehydro or hydroxylated ketamine metabolite. (2R,6R)-hydroxynorketamine The disclosure also provides novel ketamine metabolite prodrugs. The disclosure provides methods of treating, bipolar depression, major depressive disorder, neuropathic and chronic pain, including complex regional pain synthetic pain disorder (CRPD) by administering a purified ketamine metabolite or a ketamine metabolite prodrug directly to patients in need of such treatment.

Abstract: Disclosed herein are bisarylmethylthioacetamides and bisarylmethylthioethylamines useful as inhibitors of monoamine transporters. The compounds are potent and/or selective inhibitors of dopamine (DA), serotonin (5-HT), and/or norepinephrine (NE) reuptake via their respective transporters, DAT, SERT and NET. Also disclosed are methods for eliciting a wake-promoting or cognitive or attention enhancing effect and for treating substance use disorders, attention deficit (hyperactivity) disorder, depressive disorders, bipolar disorder or other neuropsychiatric disorders sleep disorders or cognitive impairment using the compounds.

Abstract: A method of carrying out adoptive immunotherapy by administering a subject an antigen-specific cytotoxic T lymphocytes (CTL) preparation in a treatment-effective amount is described. In the method, the CTL preparation is preferably administered as a preparation of an in vitro antigen-stimulated and expanded primate CTL population, the CTL population: (i) depleted of FoxP3+ T lymphocytes prior to antigen stimulation; (ii) antigen-stimulated in vitro in the presence of interleukin-21; or (iii) both depleted of FoxP3+ T lymphocytes prior to antigen stimulation and then antigen-stimulated in vitro in the presence of interleukin-21. Methods of preparing such compositions, and compositions useful for carrying out the adoptive immunotherapy, are also described.

Abstract: The present invention provides a panel of at least five clinical analyses or tests (using serum samples) to determine the risk of pediatric acute-onset neuropsychiatric syndrome (PANS) and/or pediatric autoimmune neuropsychiatric disorder associated with streptococcal infection (PANDAS) in an individual. These include enzyme linked immunosorbent assays (ELISAs) to measure antibody titers against neuronal antigens present in the brain; the neuronal antigens include lysoganglioside, tubulin, dopamine receptor D1, dopamine receptor D2, serotonin receptor 5HT2A, and serotonin receptor 5HT2C. Antibody titers against at least four of these neuronal antigens are required in the present methods; preferably antibody tiers against all of these neuronal antigens are measured. A final assay is used to quantify calcium/calmodulin-dependent protein kinase activity using a neuronal cell line.