Described herein are compositions and methods for treating Alzheimer's disease or dementia. The compositions include mammalian suppressor of taupathy 2 inhibitors (MSUT2). The MSUT2 inhibitors can be small interfering RNAs, guide RNAs, or small molecules. The methods include reducing accumulation of phosphorylated and aggregated human tau.

Provided is an animal model that can replicate a disease state of human AD. A non-human primate model animal of Alzheimer's disease includes the PSEN1 gene in which a site related to splicing of exon 9 is made deficient.

The present invention provides Factor IX fusion proteins with higher specific activity and a longer useful clotting function relative to wild type or non-modified Factor IX protein.

The present invention relates to a degenerative disease model Drosophila. More particularly, a technique is disclosed for alleviating the phenotypes of a degenerative disorder symptom of Drosophila. Using this technique, illumination with low dose radiation on a degenerative disease model Drosophila alleviates symptoms of the degenerative disease.

The present invention provides Factor IX fusion proteins with higher specific activity and a longer useful clotting function relative to wild type or non-modified Factor IX protein.

The present disclosure provides a genetically modified mouse comprising a genomic nucleic acid encoding human APOE4, a genomic nucleic acid encoding mouse TREM2 modified to include a R47H substitution, and at least one genomic modification selected from the group consisting of: (a) a genomic nucleic acid encoding mouse ABCA7 modified to include an A 1541 G substitution; (b) a genomic nucleic acid encoding mouse APP modified to include G60IR, F606Y, and R609H substitutions; (c) a genomic nucleic acid encoding mouse PLCG2 modified to include a M28L substitution; (d) a genomic nucleic acid encoding mouse MTHFR modified to include a A262V substitution; (e) an inactivated Ceacaml allele; and (f) an inactivated II1rap allele. Methods of producing the genetically modified mouse and methods of using the genetically modified mouse are also provided.

Provided herein are methods and agents for modulating the signaling pathway and components thereof that are responsible for assembly and disassembly of synapses in neurons, including amyloid beta (Aβ) mediated synaptotoxicity and synapse loss. Also provided herein are methods for screening and identifying candidate agents capable of modulating synapse formation and (Aβ) mediated synaptotoxicity.

The present disclosure provides a pharmaceutical composition for treating Alzheimer's disease and use thereof, the pharmaceutical composition containing, as an active ingredient, stem cells (SC) having high adaptability under hypoxic conditions. Particularly, the present disclosure discloses a pharmaceutical composition for alleviating or treating the symptoms of Alzheimer's disease, the pharmaceutical composition containing, as an active ingredient, mesenchymal stem cells (MSC) which have high adaptability or viability in hypoxic environments and comprise one or more knock-out HIF1AN genes.

The invention relates to animal models, and in particular to novel in vivo animal models for neurodegenerative disorders, such as Alzheimer's disease, Parkinson's disease or Motor Neurone Disease. The invention extends to methods for providing such models. The invention also provides animal models per se and methods for investigating the underlying mechanisms occurring in such neurodegenerative disorders, in particular, Alzheimer's disease, and also extends to models, methods and assays for testing pharmacological test compounds, which may modulate neurological processes, and for drug screening for use in treating neurodegenerative diseases.

The present invention relates to a method of treating a tauopathy in a subject, a method of improving cognitive ability in a subject suffering from cognitive impairment associated with a tauopathy, a method of reducing tau aggregates and neurofibrillary tangles. The method comprises administering an agent which promotes phosphorylation of tau at threonine in the sequence SSPGSPGTPGSRSR of the tau; and/or introduces a phosphomimetic of a phosphorylated tau, wherein the phosphorylated tau is tau that has been phosphorylated at threonine in the sequence SSPGSPGTPGSRSR of the tau.