The present invention relates to a composition for producing an extracellular matrix from a eukaryotic cell, the composition comprising a polypeptide or compound capable of specifically binding to a microtubule associated serine/threonine kinase family member 4 (MAST4) protein or a fragment thereof or a polynucleotide, polypeptide or compound capable of specifically binding to a nucleic acid coding for the MAST4 protein or a fragment thereof, and a composition for promoting chondrogenesis, comprising the same composition.

The present disclosure relates to human porphobilinogen deaminase derived proteins and polynucleotides and methods of using these proteins and polynucleotides.

In some aspects, the disclosure relates to compositions and methods useful for the diagnosis and treatment of diseases associated with a metabolic imbalance in a subject (e.g., cancer). In some embodiments, the methods comprise administering to a subject an N-acetylaspartate (NAA)-depleting agent or an N-acetylaspartate (NAA)-increasing agent based upon the subject's metabolic profile.

The invention relates to a method of expanding a population of regulatory T cells in a tissue or organ of a subject, wherein said method comprises administration of IL-2 and a targeting moiety specific for said tissue or organ, and wherein said tissue or organ is the central and/or peripheral nervous system. The invention further relates to populations of regulatory T cells produced according to the method and the production of said population in vivo. Also provided is a pharmaceutical composition comprising IL-2 and a targeting moiety as defined herein as well as a method of treating a disease or disorder mediated by inflammation or for the reduction of inflammation which comprises the methods defined herein or administration of a pharmaceutical composition as defined herein.

Methods and technologies for the treatment of methylmalonic acidemia.

An object of the present invention is to provide a pharmaceutical composition or food or drink composition comprising an active ingredient that suppresses functional expression of Oscar protein. Another object of the present invention is to provide a pharmaceutical composition or food composition for preventing or treating kidney disease. A further object of the present invention is to provide a pharmaceutical composition or food or drink composition that suppresses functional expression of Oscar in a living organism in order to suppress functional expression of FGF23. A still further object of the present invention is to provide a method for evaluating an effect, in the body, of an active ingredient that suppresses functional expression of Oscar protein. The above objects are achieved by at least one member selected from the group consisting of antagonists of the Oscar protein; genome editing systems that target Oscar gene; at least one RNA molecule selected from the group consisting of siRNA, shRNA, and miRNA that target Oscar mRNA, or vectors capable of expressing the RNA molecule; and antibodies that specifically bind to the Oscar protein and suppress function of the Oscar.

Targeted Factor VIII molecules comprising a Factor VIII linked with at least one domain that specifically binds to a membrane protein on a red blood cell is provided. The disclosed targeted coagulation factors prolong their duration of action and thus, are an improvement for the treatment of hematological diseases such as hemophilia A.

Compositions for and methods of treating liver injury are provided. Said compositions comprise MG53 or express MG53. Said compositions can be used for treating chronic or acute injured liver tissue and can be administered systemically, locally, or both.

A system is provided comprising a plurality of C. elegans cultures, where each culture comprises a transgenic C. elegans strain that models a mammalian disease or condition. Methods of using a system, e.g., for characterizing microbial strains of a mammalian microbiome and determining whether such microbial strains affect a mammalian disease or disorder.

Provided are compounds that target the lanthionine synthetase C-like protein 2 pathway and cells, such as immune cells, prepared in vitro with the compounds. The compounds and cells can be used to treat a number of conditions, including infectious diseases, hyperproliferative disorders, inborn errors of metabolism, chronic immunometabolic diseases, autoimmune diseases, organ transplant rejection, inflammatory disorders, and chronic pain, among others.