The present invention features compositions of Tmem100 peptides and variants thereof, and their use in treating or preventing diseases or conditions.

The present invention relates to a Crumbs homologue (CRB) therapeutic for use as a medicament or in a method of treatment or prophylaxis, for example in the treatment or prophylaxis of a retinal disorder due to mutations in the Crumbs homologue-1 (CRB1) gene, such as Leber's congenital amaurosis 8 (LCA8) or retinitis pigmentosa 12 (RP12). In particular, the present invention relates to a recombinant viral vector comprising CRB2 or modified non-toxic forms of either CRB1 or CRB3 that resemble CRB2.

Nucleic acid molecules and compositions, and methods using the same are provided herein for intraocular gene-based delivery and expression of two or more proneural bHLH transcription factors in the retina. The nucleic acid molecules, compositions and methods disclosed herein stimulate regeneration of retinal interneurons from retinal Müller glia (MG) and reprogram the MG into bipolar, amacrine, horizontal, and/or ganglion cells. Such methods and nucleic acid molecules are used for vision restoration and/or treatment of a range of ocular diseases involving retinal degeneration after injury, disease, or vison loss.

Inhibitors of miRNA-128 capable of promoting cardiomyocyte mitotic cell proliferation and methods effective for regeneration of heart tissue.

Genetically modified pigs having at least one cancer and/or at least one co-morbid condition are provided. Also provided are methods of using the pig and derived tumor cells to screen for therapeutic compounds, medical devices or procedures, and/or combinations thereof. Further provided are methods of producing personalized cancer models, including obtaining a tumor sample from a subject, identifying mutations in the tumor sample, and producing a genetically modified tumor or tumor cell line having the same mutations.

The invention provides for transgenic donor animals (e.g., pigs) whose cells, tissues and organs have a better long-term survival when transplanted into a human patient. The transgenic donor animal carries one or more human transgenes which is expressed only when the endogenous gene of the donor animal is knocked out shortly before a graft is harvested for transplantation. This “genetic switch” allows the donor animal to remain healthy during the majority of its lifetime, while still allowing expression of the human transgene for optimal transplant tolerance in a human recipient. The transgene may encode a cytokine receptor, an adhesion molecule, or a complement regulatory protein.

Two or more conditional, dominant, lethal gene expression systems provide high levels of penetrance in insects. Lethality is induced at an earlier stage of development and the risk of biochemical resistance is reduced, as compared to a single insect conditional, dominant, lethal gene expression system. The invention is useful for the control of insect populations.

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.

Provided are a targeting vector, a nucleic acid composition, and a method for constructing a liver-injured mouse model. The targeting vector includes a first expression cassette and a second expression cassette located downstream of the first expression cassette, the first expression cassette has the following elements connected in series in sequence: a liver-specific promoter, a tetracycline transcription activation regulating factor, and a first polyA; and the second expression cassette has the following elements connected in series in sequence: a second polyA, a mouse prourokinase activator encoding gene, and a tetracycline-inducible promoter. The liver-injured mouse model constructed with this targeting vector has the phenotype of spontaneously generating the liver injury and aggravating the liver injury by induction, which provides liver-injured mouse models for studies of liver diseases.

Method for controlling for the appearance of seizures in the mammalian brain comprising modifying the abundance of a specific miRNA—miR-211, for uses in preventing seizures and providing a model system to examine the effect of a drug or a treatment to seizures.