The present disclosure relates methods and compositions useful for prevention of porcine reproductive and respiratory syndrome virus (PRRSv) in animals, including animals of the species Sus scrofa. The present teachings relate to swine wherein at least one allele of a CD163 gene has been inactivated, and to specific methods and nucleic acid sequences used in gene editing to inactivate the CD163 gene. Swine wherein both alleles of the CD163 gene are inactivated are resistant to porcine reproductive and respiratory syndrome virus (PRRSv). Elite lines comprising homozygous CD163 edited genes retain their superior properties.

The present disclosure relates to a dwarfism animal model carrying an IGF-1 gene mutation and a method for generating the same. According to the present disclosure, the problem that an animal dies immediately after birth is overcome, the majority of phenotypes seen in Laron syndrome patients may be observed in the dwarfism animal model, and the dwarfism animal model has decreased expression of personality genes. Thus, the dwarfism animal model may be effectively used as a dwarfism-related disease model.

The present disclosure provides compositions and methods related to the assessment of gene editing technologies in an animal model with single-cell resolution. In particular, the present disclosure provides a novel gene editing reporter system and transgenic animal platform for testing and optimizing gene editing technologies in vivo prior to implementation in humans.

A Thy1 gene promoter specifically expressed in neurons and a recombinant vector including the Thy1 gene promoter are provided. The Thy1 gene promoter may be utilized to regulate an expression of a target gene in preparation of an animal model similar to a human.

Provided herein are genetically modified pigs, porcine organs, tissue, and cells having a reduced propensity to cause a rejection response in a human subject following xenotransplantation. In particular, provided herein are genetically modified pigs lacking nonGal xenoantigens, and porcine cells, tissues, and organs obtained from such genetically modified pigs that are suitable for transplantation into a human. Also provided herein are methods of improving a rejection related symptom in a human subject.

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.

The present disclosure relates methods and compositions useful for prevention of porcine reproductive and respiratory syndrome virus (PRRSv) in animals, including animals of the species Sus scrofa. The present teachings relate to swine wherein at least one allele of a CD163 gene has been inactivated, and to specific methods and nucleic acid sequences used in gene editing to inactivate the CD163 gene. Swine wherein both alleles of the CD163 gene are inactivated are resistant to porcine reproductive and respiratory syndrome virus (PRRSv). Elite lines comprising homozygous CD163 edited genes retain their superior properties

Described herein are methods and compositions related to vectors, including but not limited to a method for treating cystic fibrosis (CF) using adeno-associated vims (AAV) particles, using a catheter to administer a population of viral vectors to a plurality of target sites in a subject by bronchial artery catheterization delivery.

A culture medium is provided for establishing expanded potential stem cell (EPSC) lines for mammals. Methods are provided using the medium for the in vitro conversion and maintenance of cells, including pluripotent cells into EPSCs.