The present invention provides a prophylactic or therapeutic agent for a kidney disease, comprising Apoptosis Inhibitor of Macrophage (AIM) or a partial peptide thereof, or a nucleic acid comprising a base sequence encoding the same, or a screening method for a prophylactic or therapeutic agent for a kidney disease, comprising using an animal obtained by subjecting a non-human mammal deficient in AIM expression to unilateral ureteral obstruction or transient kidney ischemia/reperfusion and the like.

The inventive technology is directed to the generation of a novel transgenic mammalian model for the study of Laing distal myopathy. The novel animal model of the invention may include a transgenic animal, and preferably a transgenic mouse, expressing the β-myosin R1500P mutation transgene that produces one or more phenotypes associated with MPD1. The β-myosin R1500P mutation transgene may further be selectively expressed in fast muscle tissue of the transgenic animal.

The present invention provides an agent that enables Sirt7 gene expression, especially a recombinant adeno-associated virus (rAAV) that enables vascular endothelium (VE)-specific Sirt7 gene expression, and the use thereof. The present invention also provides a method for improving neovascularization, ameliorating aging features, extending lifespan and treating age-related diseases by using the agent, especially the rAAV.

The present invention pertains to a medicine, said medicine comprising as an active ingredient a substance which suppresses the expression of at least one molecule selected from the group consisting of TMEM5, VSTM2L, C2CDD4D, VSTM2A, LY6G6C and ADRA2C or inhibits the activity thereof, for preventing and/or treating at least one disease or symptom selected from the group consisting of progressive multiple sclerosis, gastroenteritis, myocardial disorder and sudden death. Also, the present invention pertains to a method for screening a substance, which is capable of preventing and/or treating at least one disease or symptom selected from the group consisting of progressive multiple sclerosis, gastroenteritis, myocardial disorder and sudden death, with the use of the expression or activity of the aforesaid molecule as an index.

Deferred treatment of nerve injuries is provided. Accordingly, there is provided a method of deferred treatment of a nerve injury in a subject in need thereof, the method comprising implanting at least 1 week following onset or diagnosis of the nerve injury in the subject a composition comprising a hyaluronic acid, a laminin polypeptide and an antioxidant at or near the nerve injury of the subject.

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.

The disclosure provides viral vector delivery systems for use in treating diseases or disorders in a subject to whom the viral vector delivery systems are administered, as well as to methods of making and using the viral vector delivery systems.

Described is a targeted gene therapy for use in the delay or treatment of a symptomatic stage of aging and/or age-related disease in a subject, in particular to maintain or restore endoplasmic reticulum proteostasis. The gene therapy comprises the administration of a therapeutically effective amount of a pharmaceutically acceptable composition comprising X-box binding protein 1 (XBP1) or an agent that stimulates neuronal expression of XBP1 in the brain of the subject.

The present invention provides a method of constituting a tissue construct in vitro using a tissue without depending on scaffold materials.

A method of integrating a biological tissue with a vascular system in vitro, comprising coculturing a biological tissue with vascular cells and mesenchymal cells. A biological tissue which has been integrated with a vascular system by the above-described method. A method of preparing a tissue or an organ, comprising transplanting the biological tissue described above into a non-human animal and differentiating the biological tissue into a tissue or an organ in which vascular networks have been constructed. A method of regeneration or function recovery of a tissue or an organ, comprising transplanting the biological tissue described above into a human or a non-human animal and differentiating the biological tissue into a tissue or an organ in which vascular networks have been constructed. A method of preparing a non-human chimeric animal, comprising transplanting the biological tissue described above into a non-human animal and differentiating the biological tissue into a tissue or organ in which vascular networks have been constructed. A method of evaluating a drug, comprising using at least one member selected from the group consisting of the biological tissue described above, the tissue or organ prepared by the method described above, and the non-human chimeric animal prepared by the method described above. A composition for regenerative medicine, comprising a biological tissue which has been integrated with a vascular system by the method described above.

Aspects of the disclosure relate to compositions and methods for expressing a Factor H protein (or a variant thereof) in a cell or subject. In some embodiments, the disclosure provides isolated nucleic acids and rAAVs comprising a transgene encoding a Factor H protein variant and one or more regulatory sequences. In some embodiments, compositions described herein are useful for treating subjects having diseases associated with Factor H deficiency.