Abstract: Disclosed are improved cell-permeable Parkin recombinant proteins (iCP-Parkin) which have been developed as a protein-based anti-neurodegenerative agent for efficient BBB-penetration to effectively deliver the recombinant protein into the brain. A Parkin protein, a dopaminergic neuronal cell death inhibitor, has been fused with a newly developed advanced macromolecule transduction domain (aMTD) and preferably with a solubilization domain (SD) to increase the solubility/yield and cell-/tissue-permeability of the recombinant protein. In addition, the aMTD/SD-fused recombinant iCP-Parkin protein has shown BBB-permeability. Both in vitro and in vivo, the iCP-Parkin recombinant protein improved motor skills, a typical phenotype of Parkinson's disease, by increasing dopamine level in the brain by suppressing apoptosis of dopaminergic neuron cells.

Abstract: The present invention provides a composition and method for treating, delaying the onset, delaying progression of, reducing the incidence of or reducing the severity of amyotrophic lateral sclerosis, in a subject. The composition in some embodiments of the invention is an immunoglobulin derived Fc fragment which binds and partially antagonizes the action of CD16.

Abstract: Antibodies to human N3pGlu A?, compositions comprising such N3pGlu A? antibodies, and methods of using such N3pGlu A? antibodies for the treatment of a disease characterized by deposition of A? including clinical or pre-clinical Alzheimer's disease, Down's syndrome, and clinical or pre-clinical cerebral amyloid angiopathy.

Abstract: Provided is a test method comprising a step for measuring the number of SSEA-3-positive pluripotent stem cells present in a blood sample collected from a subject, the test method providing a prognosis for cerebral infarction in the subject, and the diagnosis or prediction of asymptomatic cerebral infarction or the risk of cerebral infarction after a transient ischemic attack in the subject using the number of pluripotent stem cells as an index.

Abstract: The present invention relates to a composition for preventing or treating amyotrophic lateral sclerosis, the composition containing, as an active ingredient, two or more isoforms of a hepatocyte growth factor (HGF) or a polynucleotide encoding the isoforms. The composition of the present invention is used to effectively prevent or treat amyotrophic lateral sclerosis.

Abstract: The serotonin receptor 5HT2A (5HT2aR) and membrane NADPH oxidases (NOX enzymes) are found to be a target of autoantibodies present in Multiple Sclerosis patients. The present invention refers to peptides comprised in the extracellular regions of the human 5HT2aR and/or NOXs for diagnosis and therapy of Multiple Sclerosis.

Abstract: Compositions and methods for measuring C-peptide binding by cells, including cells expressing Glut1, using a C-peptide binding facilitator, such as an albumin. Such methods include incubating the cell with a known amount of C-peptide and a C-peptide binding facilitator, and determining the amount of C-peptide bound to the incubated cells. Also provided are methods for detecting, monitoring, and/or treating immune-mediated diseases, such as multiple sclerosis (MS), using method of the present technology for measuring C-peptide binding by cells obtained from a human or other animal subject.

Abstract: The present disclosure relates generally to the treatment of diseases and conditions exacerbated by signalling via the erythropoietin-producing-hepatoma receptor kinases EphA4 and to agents useful in such treatment.

Abstract: The invention provides antibodies, antibody drug conjugates, antibody-based fragments or antibody fragments (antigen-binding fragments), as well as antibody drug conjugates (ADCs) and chimeric antigen receptors (CARs), that specifically recognize human ROR1 and related compositions. Also provided in the invention are methods of using such antibodies in various diagnostic and therapeutic applications.

Abstract: The present invention is related to NDP-MSH or pharmaceutically acceptable salts thereof for therapeutic and/or prophylactic therapeutic treatment of inflammatory and/or neurodegenerative disorders of the CNS or multiple sclerosis. The present invention is further related to pharmaceutical compositions and a kit comprising NDP-MSH or pharmaceutically acceptable salts thereof.

Abstract: Newly identified mammalian taste-cell-specific G protein-coupled receptors, and the genes and cDNA encoding said receptors are described. Specifically, T1R G protein-coupled receptors active in taste signaling, and the genes and cDNA encoding the same, are described, along with methods for isolating such genes and for isolating and expressing such receptors. Methods for representing taste perception of a particular taste stimulus in a mammal are also described, as are methods for generating novel molecules or combinations of molecules that elicit a predetermined taste perception in a mammal, and methods for simulating one or more tastes. Further, methods for stimulating or blocking taste perception in a mammal are also disclosed.

Abstract: Embodiments described herein relate to restorative solutions for segmental peripheral nerve (PN) defects using allografted PNs for stimulating PN repair. More specifically, embodiments described herein provide for localized immunosuppression (LIS) surrounding PN allografts as an alternative to systemically suppressing a patient's entire immune system. Methods include localized release of immunosuppressive (ISV) agents are contemplated in one embodiment. Methods also include localized application of immunosuppressive (ISV) regulatory T-cells (Tregs) in other embodiments. Hydrogel carrier materials for delivery of ISV agents and are also described herein.

Abstract: The present invention is directed to a method for treating a neurodegenerative disease such as amyotrophic lateral sclerosis (ALS), Alzheimer disease, Parkinson's disease, Huntington's disease, frontotemporal degeneration, dementia with Lewy bodies, a motor neuron disease, or a demyelinating disease. The method comprises administering to a subject in need thereof a Ppargc1a activator 2-(4-tert-butylphenyl)-1H-benzimidazole, 2-[4-(1,1-dimethylethyl)phenyl]-1H-benzimidazole, in an effective amount. A preferred route of administration is oral administration.

Abstract: Disclosed herein are compositions and formulations containing a TATk-CDKL5 fusion protein. Also disclosed are methods of producing a TATk-CDKL5 fusion protein from vectors containing a TATk-CDKL5 cDNA and methods of transducing cells with the vectors containing a TATk-CDKL5 cDNA and the TATk-CDKL5 fusion protein.

Abstract: Mitofusin modulatory peptides are described which may function as activators or inhibitors of mitochondrial fusion. The sequences and compositions comprising the sequences are useful for treating diseases or disorders associated with mitofusin 1 (Mfn1) and/or mitofusin 2 (Mfn2) and mitochondrial dysfunction. Methods of treatment, pharmaceutical formulations and methods of identifying compounds that mimic the activity of the peptides for use in screening assays are also described.

Abstract: The disclosure provides nucleic acids, including cDNA, comprising alterations that encode aspartic acid at a position corresponding to position 533 of the human G protein-coupled receptor 156 protein (GPR156). The disclosure also provides isolated and recombinant human GPR156 protein variants that comprise an aspartic acid at a position corresponding to position 533. The change to aspartic acid, and the gene encoding this change, associate with unipolar depression. The disclosure also provides methods for determining whether a subject has or has a risk of developing unipolar depression, based on the identification of such alterations in the gene (DNA or RNA) encoding GPR156.

Abstract: The present invention provides a composition and method for treating, delaying the onset, delaying progression of, reducing the incidence of or reducing the severity of amyotrophic lateral sclerosis in a subject. The composition a peptide derived from SOD1 which can be used to inhibit formation of SOD1 amyloid-like aggregates or for production of anti-SOD1 antibodies.

Abstract: Methods for treating, or reducing risk of developing, seasonal worsening of multiple sclerosis (MS) in a subject who has MS, comprising administering a melatonin agonist to a subject.

Abstract: The present invention provides a skin penetration enhancing method and its penetration enhancer which is comprised of sponge spicules or a biologically effective amount of sponge spicules or a combination containing sponge spicules. The penetration enhancer can be applied onto the skin to overcome the skin stratum corneum barrier, which can increase the percutaneous absorption of drugs, vaccines and cosmetics into different skin layers or can improve the transdermal delivery of all these therapeutics into the systemic blood circulation. This technology can be used for dermal delivery of therapeutics into the skin or for transdermal delivery of therapeutics across the skin into systemic blood circulation.

Abstract: The present invention relates to mutant transmembrane proteins which have increased conformational stability when compared to their parent protein, methods of selection and production. In particular the invention relates to mutant transmembrane proteins which are mutated in or in the proximity of the transmembrane alpha helices or in a kinked region or in an alpha-helix adjacent to a kink. The mutant transmembrane proteins have use in crystallisation studies and also in screening to identify compounds for use in drug discovery and therapy.