The present invention relates to SorCS1-like agents, including SorCS1, nucleic acid molecule encoding expression of SorCS1 and fragments thereof, as well as vectors containing said nucleic acid and to cells expressing SorCS1 and said fragments, for the treatment of obesity.

The present invention provides isolated promoters, transgene expression cassettes, vectors, kits, and methods for treatment of genetic diseases that affect the cone cells of the retina.

Embodiments of the present invention describe a method of changing the phenotype of monocytes and macrophages from a proinflammatory M1 phenotype to an anti-inflammatory M2 phenotype. The method can comprises providing a composition comprising a recombinant adeno-associated virus (rAAV) vector comprising an exogenous gene encoding ApoA-I Milano or a fragment thereof, and administering the composition to a mammal in need thereof to change the phenotype of monocytes or macrophages from a proinflammatory M1 phenotype to an anti-inflammatory M2 phenotype. By changing the phenotype of monocytes or macrophages from a proinflammatory M1 phenotype to an anti-inflammatory M2 phenotype, atherosclerosis can be treated. The present invention also describes a method of monitoring macrophage phenotypic switching and a method of assessing the efficacy of the treatment of atherosclerosis.

The present invention relates to a polynucleotide configured for the treatment of a disease of retinal cone cells, such as achromatopsia, a nucleic acid vector comprising said polynucleotide, a pharmaceutical composition comprising said nucleic acid vector, a kit comprising said polynucleotide or said nucleic acid vector, a method of making said nucleic acid vector, and a method for treating a disease of the retinal cone cells.

Described are nucleic acid regulatory elements that are able to enhance liver-specific expression of genes, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. These are particularly useful for applications using gene therapy.

The present invention provides reagents and methods for modulating cone photoreceptor activity, and devices for assessment of cone photoreceptor activity.

An adeno-associated viral (AAV) vector containing an expression construct, wherein: the expression construct comprises a nucleic acid sequence which encodes a microdystrophin (MD); and the nucleic acid sequence encoding the MD has a size of at least 4.1 kb.

Provided herein are animals expressing light-responsive opsin proteins in the basal lateral amygdala of the brain and methods for producing the same wherein illumination of the light-responsive opsin proteins causes anxiety in the animal. Also provided herein are methods for alleviating and inducing anxiety in an animal as well as methods for screening for a compound that alleviates anxiety in an animal.

Expression of a phosphatase inhibitor in heart cells can be used to treat cardiac disorders, e.g., heart failure. Decreasing phosphatase activity can improve -adrenergic responsiveness.

Recombinant, chimeric porcine influenza viruses are disclosed that include hemagglutinin segments from more than one influenza virus subtype. Also described are methods of producing the recombinant influenza viruses, immunogenic compositions comprising the recombinant influenza viruses, methods of stimulating an immune response against influenza virus, and methods of treating and preventing influenza virus infection.