This disclosure relates to an animal model of human disease. More specifically, this disclosure relates to a rodent model of mood disorders such as unipolar depression and an anxiety disorder. Disclosed herein are genetically modified rodent animals that carry a humanized G protein-coupled receptor 156 (GPR156) gene that encodes a mutant human GPR156 protein comprising Asp at an amino acid position corresponding to position 533 in a full length wild type human GPR156 protein.

The present invention relates to an AAV vector carrying a predetermined hybrid HGF gene sequence. Use of the AAV vector of the present invention allows a hybrid HGF gene to be delivered to a subject at a high delivery yield.

Non-human animal cells and non-human animals comprising CRISPR/Cas synergistic activation mediator system components and methods of making and using such non-human animal cells and non-human animals are provided. Methods are provided for using such non-human animals to increase expression of target genes in vivo and to assess CRISPR/Cas synergistic activation mediator systems for the ability to increase expression of target genes in vivo.

This disclosure relates to an animal model of human disease. More specifically, this disclosure relates to a rodent model of mood disorders such as unipolar depression and an anxiety disorder. Disclosed herein are genetically modified rodent animals that carry a humanized G protein-coupled receptor 156 (GPR156) gene that encodes a mutant human GPR156 protein comprising Asp at an amino acid position corresponding to position 533 in a full length wild type human GPR156 protein.

The present disclosure encompasses methods for generating cells or tissue from existing cells with one or more mutated variants of Yap. In specific embodiments, the disclosure regards treatment of existing cardiomyocytes with one or more mutated variants of Yap that causes them to divide and generate new cardiomyocytes. In specific cases, the mutated variant of Yap has serine-to-alanine substitutions at 1, 2, 3, 4, 5, 6, or more serines of Yap.

Provided herein are adeno-associated virus (AAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cell. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.

The present disclosure relates to genetically modified animals and cells with humanized heavy chain immunoglobulin locus and/or humanized light chain immunoglobulin locus.

Compositions including modified adeno-associated virus (AAV) vectors comprising a recombinant AAV (rAAV)-based genome are provided herein, wherein the rAAV-based genome includes one or more of: a brain derived neurotrophic factor (BDNF)-encoding cDNA insert; or a neurotrophin-3 (NT-3)-encoding cDNA insert. Also provided are methods of treating motor neuron degenerative disorders, such as amyotrophic lateral sclerosis (ALS), by administering the disclosed compositions.

Provided are compositions for simultaneously modifying amino acids of site 736 and site 738 of pAPN gene and application thereof. An sgRNA set specifically recognizing porcine pAPN gene can recognize sequences near amino acids of site 736 and site 738 of pAPN gene, and has strong specificity. On this basis, in combination with a composition consisting of a cleavage protein and a double-stranded donor sequence, simultaneous modification of amino acids of site 736 and site 738 of the pAPN gene can be realized. Under editing of the composition, the amino acids of site 736 and site 738 of the pAPN gene are modified precisely and effectively, which can prevent normal expression of other amino acids of the pAPN gene from being damaged or changed. Therefore, it can resist TGEV infection, and also can retain the physiological activity function of the pAPN protein to the maximum extent.

A method of inhibiting an overactive fibroblast growth factor receptor 3 (FGFR3) in a cell by contacting the cell with a composition that contains an effective amount of Pheophorbide a, Pyropheophorbide a, or an active derivative thereof. Also disclosed is a method for treating a disorder associated with an overactive FGFR3 with a composition containing an effective amount of Pheophorbide a, Pyropheophorbide a, or an active derivative thereof. Further, a composition for treating a disorder associated with an overactive FGFR3 is described. The composition contains an ethanol extract of Amaranthus viridis.