A method and composition to treat a subject with arginase 1 (ARG1) deficiency (ARG1-D) and to rapidly reduce the levels of at least one of arginine and/or a guanidino compound in the subject.

The present invention relates to the combination of spirulina and palmitoylethanolamide (PEA) and/or pharmaceutically acceptable derivates or salts thereof, pharmaceutical formulations comprising the combination of spirulina and PEA and/or pharmaceutically acceptable derivates or salts thereof, optionally together with at least one physiologically acceptable excipient, and the use of the combination of spirulina and PEA and/or pharmaceutically acceptable derivates or salts thereof and formulations which include the said combination, for use in the prevention and/or treatment of tissue hyperactivation states, in the prevention and/or treatment of inflammatory pathologies, in the prevention and/or treatment of alterations in cardiac and/or coronary tissue, in the prevention and/or treatment of alterations in the vascular tissue, in the prevention and/or treatment of ophthalmic pathologies, preferably in the prevention and/or treatment of macular degeneration pathologies and glaucoma, in the prevention and/or treatment of dyslipidemia, in the prevention and/or treatment of alterations in pulmonary tissue, in the prevention and/or treatment of alterations in pelvic tissues, in the prevention and/or treatment of cellular alterations due to carcinogenesis, and/or in the prevention and/or treatment of dermatological alterations.

Combining viral vector with surfactant preserves vector infectivity, and surfactant provided an unexpected benefit by protecting viral vector from damage due to transient elevated temperature.

Compounds useful as biologically active compounds are disclosed. The compounds have the following structure (I): ##STR00001##
or a stereoisomer, tautomer or salt thereof, wherein R.sup.1, R.sup.2, R.sup.3, L, L.sup.1, L.sup.2, L.sup.3, M and n are as defined herein. Methods associated with preparation and use of such compounds is also provided.

The invention relates to surface modified extracellular vesicles, wherein the extracellular vesicles comprise an exogenous polypeptide tag that is covalently linked to a membrane protein of the extracellular vesicles. In a particular embodiment, the tag is covalently linked to the membrane protein of microvesicles by sortase-mediated ligation. Methods of preparing said extracellular vesicles and methods of using said extracellular vesicles loaded with therapeutic molecules for treating a disease are also disclosed herein.

The present invention relates to a method for preparing viral vector-based compositions wherein the viral vector-based particles present in the composition have a particle size distribution with a polydispersity index (PDI) of less than 0.5, the method comprising the steps: (a) providing replication-deficient viral vectors; (b) providing a solution comprising at least one sugar and at least three different excipients selected from hydrophilic and amphiphilic excipients, wherein the excipients are characterized by polar, aliphatic, aromatic, negatively charged, and/or positively charged functional groups, and wherein the solution is further characterized by an excipient-sugar ratio of at least 1:2 (w/w); and (c) mixing the replication deficient viral vectors of step (a) with the solution of step (b). The present invention further relates to a viral vector-based composition obtainable by the method of the invention as well as to the viral vector-based composition of the invention for use as a prime-boost vaccine.

Embodiments concern methods and compositions for treating or preventing a bacterial infection, particularly infection by a Staphylococcus bacterium. Aspects include methods and compositions for providing a passive immune response against the bacteria. In certain embodiments, the methods and compositions involve an antibody that binds Coagulase (Coa). Further aspects relate to immunogenic compositions comprising at least one Staphylococcal coagulase R Domain, wherein the R Domain is 80% identical in sequence to a R Domain.

Provided are a pharmaceutical composition comprising a mineralocorticoid receptor antagonist and use thereof. When the pharmaceutical composition is orally administered to a patient having chronic kidney disease in need thereof, the effective and safe AUC ranges from 188 ng*h/mL to 3173 ng*h/mL, with bioavailability of 50% or more in mammals. When the pharmaceutical composition is orally administered at a daily dose of 0.1 to 1.0 mg to treat chronic kidney disease, the AUC is controlled at a safe and effective level.

Provided are a pharmaceutical composition comprising a mineralocorticoid receptor antagonist and use thereof. When the pharmaceutical composition is orally administered to a patient having chronic kidney disease in need thereof, the effective and safe AUC ranges from 188 ng*h/mL to 3173 ng*h/mL, with bioavailability of 50% or more in mammals. When the pharmaceutical composition is orally administered at a daily dose of 0.1 to 1.0 mg to treat chronic kidney disease, the AUC is controlled at a safe and effective level.

The invention provides compositions and methods for sequencing nucleic acids and other applications. In sequencing by synthesis, unlabeled reversible terminators are incorporated by a polymerase in each cycle, then labeled after incorporation by binding to the reversible terminator a directly or indirectly labeled antibody or other affinity reagent.