Nonsense-mediated mRNA decay (NMD) polypeptides, nucleic acids encoding NMD polypeptides, and methods of using such polypeptides and nucleic acids in the treatment of ALS and in screening for agents for the treatment of ALS are described.

The present invention relates to a composition comprising a peptide derived from myelin basic protein (MBP) peptide for use in treating or preventing uveitis in a subject

Antibodies and antigen binding fragments thereof that specifically recognize and bind an epitope of elastin that is exposed and accessible in degraded elastic fiber are described. The antibodies and/or antigen binding fragments can be operably linked to a secondary component, including biologically active agents such as therapeutics and/or imaging agents. Optionally, the antibodies and/or antigen binding fragments thereof can be attached to a surface of a carrier, such as a particle, for specific binding and delivery of the carried agents to degraded elastic fiber.

A variant neurodegenerative disease-associated protein, which comprises an amino acid sequence comprising a deletion, substitution or addition of one or several amino acids present in the interaction region of two molecules of protofilaments (PF) in the amino acid sequence of a neurodegenerative disease-associated protein, and in which seed activity that functions as a nucleus of an aggregate of the neurodegenerative disease-associated protein is reduced.

The disclosure includes non-naturally occurring or engineered CRISPR Cas9, each associated with at least one destabilization domain (DD), along with compositions, systems and complexes involving the DD-CRISPR Cas9, nucleic acid molecules and vectors encoding the same, delivery systems involving the same, uses therefor.

Described herein are oligonucleotides (e.g., RNAi oligonucleotides) containing sense and antisense strands for targeting complement factor B (CFB) mRNA. The RNAi oligonucleotide may be used to inhibit CFB expression, levels, and/or activity in a cell. Also, described herein are methods for using an oligonucleotide (e.g., an RNAi oligonucleotide) for the prophylaxis or treatment of a disease, disorder, or condition mediated by complement pathway activation or dysregulation.

The present disclosure pertains to the field of animal disease models, specifically to a method for constructing an animal model of open-angle glaucoma. In this model, the pathogenesis of open-angle glaucoma is mimicked by inducing necroptosis in trabecular meshwork cells through the injection of IFN- into the anterior chamber. This process leads to the inhibition of trabecular meshwork activity and functional impairment, resulting in elevated intraocular pressure and subsequent optic nerve changes indicative of glaucoma. The pathophysiological alterations observed in the animal model closely resemble those seen in human open-angle glaucoma, including chamber angle opening, trabecular meshwork dysfunction, increased intraocular pressure, and loss of RGCs.

The disclosure relates to a sarcoidosis animal model and methods of inducing sarcoidosis in an animal. The disclosure also relates to methods of producing an in vitro granuloma, and methods of using the sarcoidosis animal model. Disclosed herein are animals comprising one or more granulomas, wherein the one or more granulomas comprise Mycobacterium abscessus cell wall microparticles.

The present invention relates to a method for producing a nonhuman primate animal model of cerebral infarction, comprising administering endothelin to basal ganglia and thalamic region of a nonhuman primate, and thereby inducing basal ganglia damage, thalamus damage, and internal capsule damage; and a pharmaceutical composition for the treatment of cerebral infarction at a subacute to chronic stage, penetrating branch infarction, or cerebral infarction having brain damage in a penetrating branch territory, comprising a NeuroD1 protein or a polynucleotide encoding the NeuroD1 protein.