The disclosure provides for single chain variable fragments to oxidized phospholipid epitopes and methods of use thereof, including the production of transgenic animal models and the use of the fragments as therapeutic agents for treating CAS.

Non-human animal models of non-alcoholic fatty liver disease (NAFLD) are provided. Compositions and methods for producing the non-human animal models and uses of the non-human animal models to screen and evaluate agents for treating or preventing NAFLD are also provided.

The present invention belongs to the technical field of protein and genetic engineering, and specifically discloses use of an erythropoietin human hepatocyte receptor B4 as a target in screening and preparing a biological formulation or medicament for increasing sensitivity to insulin. Also disclosed is use of an erythropoietin human hepatocyte receptor B4 in preparing an insulin-sensitized mouse model. On the basis of insulin signal regulation, a protein EphB4 capable of interacting with an insulin receptor (InsR) is found. The protein can interact with InsR, and insulin stimulation can promote the interaction between the two, which provides a basis for insulin resistance in the case of hyperinsulinaemia. Over-expression of EphB4 can promote degradation of InsR. Inhibition of EphB4 can enhance the sensitivity to insulin and improve insulin resistance.

The present invention relates to vectors and compositions for the treatment of glycogen storage disease III.

Regimens useful in reducing the frequency of apheresis in a human patient having familial hypercholesterolemia are described. The method involves administering to the human subject via a peripheral vein by infusion of a suspension of replication deficient recombinant adeno-associated virus (rAAV).

The present invention relates to expression constructs and vectors for the treatment and/or prevention of diseases that are associated with a loss of NPC1 function, such as the lysosomal storage disorder Niemann-Pick type C (NPC) disease.

A therapeutic regimen useful for treatment of GM1 gangliosidosis comprising administration of a recombinant adeno-associated virus (rAAV) vector having an AAV capsid and a vector genome comprising a sequence encoding human β-galactosidase is provided. Also provided are compositions containing a rAAV vector and methods of treating GM1 gangliosidosis in patient comprising administration of a rAAV vector.

The disclosure provides thiol-containing alkyl fatty acid and vitamin D compound formulations for intravenous, parenteral or oral administration. The compositions of the present technology have optimal controlled bioavailability and are useful for treating metabolic dysfunctions such as pre-diabetes, Metabolic Syndrome and diabetes. Also provided are methods of treatment comprising the daily administration of the disclosed thiolcontaining alkyl fatty acid formulations alone or in combination with the disclosed vitamin D compound formulations.

Complexes comprising a nucleic acid-guided endonuclease, a sequence-specific targeting nucleic acid and an amphipathic helical peptide are provided. Compositions and methods for delivery of complexes comprising a nucleic acid-guided endonuclease, a sequence-specific targeting nucleic acid and an amphipathic helical peptide to mammals for both research and therapeutic use are provided. Methods of treating or reducing one or more symptoms of type 2 diabetes, prediabetes and/or gestational diabetes are provided.

The present disclosure is directed to novel methods of treating type-1 or type-2 diabetes by inactivating TLR2 and TLR4 genes together in cells capable of producing insulin and/or regenerating β cells, and providing the cells to a subject in need thereof.