The present disclosure is directed, in some embodiments, to compositions and methods for inducible modification of a cell genome.

A transgenic mouse that is engineered by one or more genetic modifications that delete endogenous Adam6a and Adam6b genes in a male mouse, which mouse comprises in its genome only one exogenous Adam6 transgene, and expresses an ADAM6 protein comprising at least 90% sequence identity to SEQ ID NO:1 or SEQ ID NO:2, which Adam6 transgene is functional in a male mouse.

Livestock animals and progeny thereof comprising at least one edited chromosomal sequence that alters expression or activity of a somatostatin receptor (SSTR) protein are provided. Livestock animal cells that contain such edited chromosomal sequences are also provided. The livestock animals have improved growth performance and weight gain. Methods for producing livestock animals with increased growth performance are also provided.

Disclosed are Syntaxin 4-overexpressing pancreatic islet -cells, methods of making these cells, and methods of using these cells for promoting and protecting functional -cell mass, thereby to treat and/or prevent various insulin-related diseases and conditions including diabetes and pre-diabetes. Also disclosed are methods of treating or preventing insulin-related diseases by overexpressing Syntaxin-4.

Provided are a psoriasis-induced transgenic animal model overexpressing the Pellino homolog 1 (Peli1) gene according to doxycycline administration, and a use thereof. The transgenic animal model of the present disclosure exhibited similarity to phenotypes shown in patients with psoriasis, due to overexpression of the Pellino homolog 1 (Peli1) gene according to doxycycline administration. It is anticipated that the transgenic animal model may be usefully used in clinical studies, such as screening for a candidate drug for the treatment of psoriasis. Additionally, it is anticipated that a peptide derived from the Peli1 FHA domain targeting the FHA binding motif that inhibits normal substrate binding between a substrate protein and the Peli1 protein may be usefully used in the development of new drugs for psoriasis-associated diseases. Moreover, by confirming an expression level of the Peli1 protein, it is anticipated to be usefully used in evaluating the severity of patients with psoriasis.

The invention is directed to compositions and methods for treating or reducing the likelihood of the development of epilepsy in an individual. The method comprises administering to the central nervous system of an individual in need of such treatment a therapeutically effective amount of an agent capable of increasing the expression and/or activity of miR-128.

Provided herein are methods and compositions for treating an eye disorder, for example CORD6. Aspects of the disclosure relate to knocking out an autosomal dominant mutant GUCY2D gene.

The subject invention pertains to transgenic non-human animals comprising a transgenic nucleotide sequence, integrated into the genome of the animals, comprising a nucleotide sequence encoding human FKBP51 operably linked to a tetracycline response element. In some embodiments, the transgenic animal comprises an additional transgenic nucleotide sequence, integrated into the genome of the animal, comprising a nucleotide sequence encoding a tetracycline transactivator (tTA) operably linked to a promoter; wherein the tTA is expressed upon activation of the promoter and binds the tetracycline response element, thereby causing expression of FKBP51. The invention also pertains to methods for screening for agents for the prevention and/or treatment of psychiatric disorders, such as depression.

Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized coagulation factor XII (F12) locus and methods of making and using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized F12 locus express a human coagulation factor XII protein or a chimeric coagulation factor XII protein, fragments of which are from human coagulation factor XII. Methods are provided for using such non-human animals comprising a humanized F12 locus to assess in vivo efficacy of human-coagulation-factor-XII-targeting reagents such as nuclease agents designed to target human F12. A short isoform of F12 that is produced locally in the brain, and methods of using the short isoform, are also provide.

The invention relates to a vector comprising: a 5 nucleic acid that is homologous to a genomic sequence 5 of a stop codon of a constitutively expressed gene; an exogenous nucleic acid; a 3 nucleic acid that is homologous to a genomic sequence 3 of the stop codon of the constitutively expressed gene; a translation interruption-reinitiation signal operably linked to the 5 nucleic acid and the exogenous nucleic acid, wherein the translation interruption-reinitiation signal is capable of replacing the stop codon of the constitutively expressed gene.