The invention provides compositions and methods for effective lysosomal targeting mediated by SORT1. In particular, the compositions and methods provided by the invention may be used to treat lysosomal storage diseases such as Sanfilippo syndrome type B.

The invention provides composition comprising a fragment, derivative or variant of sortilin, or a sortilin mimetic, for use in medicine, wherein the sortilin mimetic is not SorCS1, SorCS2, SorCSS or SorLA. The composition preferably for use in treating or preventing diabetes mellitus, insulin resistance, obesity, metabolic disorders, polycystic ovary syndrome, non-alcoholic fatty liver disease, retinopathy, neuropathy, nephropathy, Alzheimer's disease and/or cardiovascular disease in a subject in need thereof. Also provided are isolated polypeptides, nucleic acids encoding the polypeptides, vectors, host cells and expression systems containing the nucleic acids and methods for making the polypeptides.

Neurokinin-1 (NK-1) receptor-binding agent delivery conjugates, compositions comprising NK-1 receptor-binding agent delivery conjugates, and methods for making and administering NK-1 receptor-binding agent delivery conjugates are provided. A conjugate may include an NK-1 receptor-binding moiety, a linker group containing at least one linker selected from the group of a releasable linker and a spacer linker, and an active agent linked to the linker group. The active agent may be selected from the group of fluorophore-containing compounds, radionuclide-containing compounds, and therapeutic agents for treatment of tumor cells characterized by over-expression of the NK-1 receptor.

The present application relates to novel peptide-based compounds, optionally comprising an immunoactive built-in adjuvant, compositions comprising these compounds and their use, in particular for the treatment of diseases, disorders or conditions characterized by or associated with β-amyloid accumulation. In particular, the present application includes compounds of Formula I, and compositions and uses thereof: [R.sup.a-NP].sub.m-L.sub.p (I).

The present disclosure relates to methods and pharmaceutical compositions for the treatment of prostate cancers. In particular, the present invention relates to an OX1R agonist for use in the treatment of prostate cancer in a subject in need thereof.

Described herein are methods of treating or preventing a fungal infection by administering one or more compounds of the present invention to a subject. The methods of the present invention treat or prevent a fungal infection of C. neoformans, C. gattii, L. prolificans, C. albicans, or a combination thereof, as examples. The compounds of the present invention may be administered to a subject with other agents such as antifungal agents.

This document relates to materials and methods for controlling ligand gated ion channel (LGIC) activity. For example, modified LGICs including at least one LGIC subunit having a modified ligand binding domain (LBD) and/or a modified ion pore domain (IPD) are provided. Also provided are exogenous LGIC ligands that can bind to and activate the modified LGIC, as well as methods of modulating ion transport across the membrane of a cell of a mammal, methods of modulating the excitability of a cell in a mammal, and methods of treating a mammal having a channelopathy.

This document relates to materials and methods for controlling ligand gated ion channel (LGIC) activity. For example, modified LGICs including at least one LGIC subunit having a modified ligand binding domain (LBD) and/or a modified ion pore domain (IPD) are provided. Also provided are exogenous LGIC ligands that can bind to and activate the modified LGIC, as well as methods of modulating ion transport across the membrane of a cell of a mammal, methods of modulating the excitability of a cell in a mammal, and methods of treating a mammal having a channelopathy.

Provided, inter alia, are methods and compositions for treating epilepsy. In one aspect, provided herein is a method of selecting a compound for treating epilepsy, said method includes, contacting a test compound with 5-hydroxytryptamine-2B receptor (5-HT2B), and measuring the 5-HT2B agonistic activity of the test compound. In another aspect, provided herein is a method of treating an epilepsy in a subject in need thereof. The method includes administering to said subject an effective amount of a 5-HT2B specific receptor agonist.

A newly developed butane-tetraol-based amphiphilic compound, a method of preparing the same, and a method of extracting, solubilizing, stabilizing, crystallizing or analyzing a membrane protein using the amphiphilic compound are provided. The butane-tetraol-based compound is found to have a central structure exhibiting chirality, and isomers of the compound have clearly different characteristics according to the stereochemistry of the central structure, thereby making it possible to select compounds suitable for the uses thereof. Also, the compound can be used to effectively extract a membrane protein, which has more various structures and characteristics than conventional compounds, from cell membranes and stably store the membrane protein in an aqueous solution for a long time, and thus analyze the function and structure of the membrane protein. The analysis of the structure and function of the membrane protein is one of the fields which have received the most attention in biology and chemistry since the analysis of the structure and function of the membrane protein is closely associated with the development of new drugs.